{"paper_id":"8c17ad12-ce6b-4b5c-bdb6-d2ff73525fc9","body_text":"Future Oncology\nISSN: 1479-6694 (Print) 1744-8301 (Online) Journal homepage: www.tandfonline.com/journals/ifon20\nTransfusion-related cost offsets and time burden\nin patients with myelofibrosis on momelotinib vs.\ndanazol from MOMENTUM\nLucia Masarova, Srdan Verstovsek, Tom Liu, Sumati Rao, Gautam Sajeev,\nMirko Fillbrunn, Ryan Simpson, Weilong Li, Joseph Yang, Yvette Le Lorier,\nBoris Gorsh & James Signorovitch\nTo cite this article: Lucia Masarova, Srdan Verstovsek, Tom Liu, Sumati Rao, Gautam Sajeev,\nMirko Fillbrunn, Ryan Simpson, Weilong Li, Joseph Yang, Yvette Le Lorier, Boris Gorsh &\nJames Signorovitch (2024) Transfusion-related cost offsets and time burden in patients\nwith myelofibrosis on momelotinib vs. danazol from MOMENTUM, Future Oncology, 20:30,\n2259-2270, DOI: 10.1080/14796694.2024.2368450\nTo link to this article:  https://doi.org/10.1080/14796694.2024.2368450\n© 2024 The Author(s). Published by Informa\nUK Limited, trading as Taylor & Francis\nGroup\nView supplementary material \nPublished online: 29 Jul 2024.\n Submit your article to this journal \nArticle views: 1825\n View related articles \nView Crossmark data\n Citing articles: 14 View citing articles \nFull Terms & Conditions of access and use can be found at\nhttps://www.tandfonline.com/action/journalInformation?journalCode=ifon20\n\nFUTURE ONCOLOGY \n2024, VOL. 20, NO. 30, 2259–2270 \nhttps://doi.org/10.1080/14796694.2024.2368450 \nRAPID COMMUNICATION \nTr ansfusion-r ela t ed cost offsets and time burden in patients with myelofibrosis \non momelotinib vs. danazol from MOMENTUM \nLucia Masarova * , a \n , Srdan Verst ov sek a , Tom Liu b , Sumati Rao b , Gautam Sajeev c , Mirko Fillbrunn c , Ryan \nSimpson c , Weilong Li c , Joseph Yang b , Yvette Le Lorier ‡, b , Boris Gorsh ‡, b and James Sig norovit ch c \na Department of Leukemia , Division of Cancer Medicine , The University of Texas MD Anderson Cancer , Houston , TX 77030, USA ; \nb GSK plc , Philadelphia , PA 19104, USA ; c Analysis Group , Boston , MA 02199, USA \nARTICLE HISTORY \nReceived 4 April 2024 \nAc c ept ed 12 June 2024 \nKEYWORDS \nACVR1; anemia; cost; \ndanazol; JAK inhibitor; \nmomelotinib; \nmy elofibr osis; time \nburden; transfusion \nABSTRACT \nA im: To estimat e project ed US-based c ost and time burden for pa tien ts with my elofibr osis \nand anemia treated with momelotinib compared with danazol. \nM etho ds: Cost and time burden were calculated based on the transfusion status of patients in the \nMOMENTUM trial and estimates ex trac ted from previous studies. \nResults: Reductions in transfusion associated with momelotinib are project ed t o result in \ncost and time savings compared with danazol in transfusion-dependent and transfusion- \nindependen t/requiring pa tien ts with my elofibr osis, r espectiv ely: annual medical costs ($53,143 and \n$46,455 per person), outpa tien t transfusion costs ($42,021 and $8,370 per person) and annual time \nsavings (173 and 35 h per person). \nConclusion: Fewer transfusions with momelotinib are projected to result in cost and time savings in \npa tien ts with my elofibr osis and anemia compared with danazol. \nPL AIN L ANGUAGE SUMMARY \nEstimated cost & time savings in patients with the blo o d cancer myelofibrosis \nMy elofibr osis is a rare blood cancer often associated with bone marrow damage, too few of \nsome types of blood cells and symptoms including tiredness, night sweating, itching and feelings \nof fullness and pain because of increased spleen size. Pa tien ts with anemia (too few red blood \ncells) may r equir e r egular blood transfusions and this is one sign that my elofibr osis is getting w orse. \nMOMENTUM was a Phase III clinical trial showing that the drug momelotinib was safe and effective \nin pa tien ts with my elofibr osis who w er e pr eviously tr eat ed with a type of drug called a JAK inhibit or. \nIn particular, the trial showed that momelotinib reduced the need for transfusions compared with \ndanazol, another drug typically used to treat patients with anemia. Based on this transfusion \ninformation from MOMENTUM and other publicly available information about estimated medical \ncosts and pa tien ts’ time spen t in rec eiving transfusions, the analy sis described here show s that a \nreduction in the number of transfusions with momelotinib compared with danazol is estimated to \nlead t o c ost savings as well as reduced pa tien t time spen t in transfusion-rela ted trav el , pr eparing and \nwaiting for transfusions and receiving and recovering from transfusions. \nTWEETABLE ABSTRACT \nReductions in transfusions associated with momelotinib ar e pr oject ed t o result in savings in medical \nand transfusion-relat ed c osts and time burden relative to danazol in pa tien ts with my elofibr osis and \nanemia. \n1. Background \nMy elofibr osis (MF) is a chr onic , pr ogr essiv e, Philadelphia \nchr omosome–negativ e my elopr oliferativ e neoplasm \n(MPN) characteriz ed b y anemia, splenomegaly, \ndebilitating symptoms and bone marrow fibrosis [ 1 ]. \nMPNs ar e rar e, with MF estimat ed t o hav e a w orldwide \nannual incidenc e rat e of 0.47 t o 1.98 per 100,000 and a \nprevalenc e rang ing from 1.76 to 4.05 per 100,000 [ 2 , 3 ]. \nWhile MF is often a primary disease, it may also \noccur post polycythemia vera (PV) or post essential \nthrombocythemia (ET) [ 1 ]. Primary MF is more aggressive \nCONTACT Lucia Masarova Tel.: + 1 855 707 8878; lmasarova@mdanderson.org \n‡Former employee of GSK \nThis article has been corrected with minor changes. These changes do not impact the academic con ten t of the article. \nSupplemental data for this article can be accessed at https:// doi.org/ 10.1080/ 14796694.2024.2368450 \n©2024 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group \nThis is an Open Ac c ess article distributed under the terms of the Cr eativ e Commons A ttribution-NonCommer cial-NoDerivativ es License \n( http:// creativecommons.org/ licenses/by- nc- nd/ 4.0/ ), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly \ncited, and is not alter ed , transformed , or built upon in any wa y. T he t erms on which this article has been published allo w the posting of the Ac c ept ed Manuscript in a reposit ory b y the \nauthor(s) or with their c onsent . \n\n2260 L. MASAROVA ET AL. \nand has a poorer prognosis than PV or ET [ 4 ], as disease \npr ogr ession with MF can include bone marr ow failur e, \ntransformation t o acut e myeloid leukemia and early \nmortality [ 5 ]. While MF can occur at any age, it is most \nfrequently diagnosed in older pa tien ts, with a median \nage of diagnosis between 65 and 70 years [ 6 , 7 ]. \nAmong pa tien ts with MF, one-thir d hav e anemia and \none-quarter have transfusion dependent (TD) anemia by \nthe time of MF diagnosis [ 8 , 9 ]. Over time, most pa tien ts \nwith MF develop anemia, which is associated with poor \nprognosis [ 9–11 ]. Any grade of anemia adversely affects \nthe survival of patients with MF, and sev er e anemia is \nassociated with a > 1.5-fold increase in the risk of death \nc ompared with moderat e anemia [ 12 ]. Ac c ording t o a \nr etr ospectiv e analysis of 1109 pa tien ts with primary MF, \nmedian survival was 4.9, 3.4 and 2.1 years for patients \nwith mild (hemoglobin [Hb] ≥10 g/dl but below the sex- \nadjusted lower limit of normal), moderate (Hb between \n8 g/dl and < 10 g/dl) and sev er e (Hb < 8 g/dl or TD) \nMF-associated anemia, r espectiv ely [ 12 ]. W hile the emer - \ngence of JAK inhibitors has provided substantial benefit \nfor splenomegaly and c onstitutional sympt oms, and may \nimpr ov e survival [ 13 ], anemia remains a challenge in MF \nmanagement . A mong curr ently appr ov ed JAK inhibitor \ntherapies for MF, ruxolitinib and fedratinib are intrinsically \nmy elosuppr essiv e and can cause or worsen anemia and \nexac erbat e transfusion dependency [ 8 , 14 ]. \nRed blood cell (RBC) transfusions are the main \ntrea tmen t option for worsening or severe anemia in \npa tien ts with MF. How ev er, transfusion dependence \nis associated with poor survival and may add to the \neconomic burden on pa tien ts, their caregivers and the \nhealth sy st em via healthcar e r esour ce utilization (HCRU) \nand costs associated with transfusions and managing \ntransfusion complications [ 15–18 ]. In a r etr ospectiv e \nanaly sis, transfusion dependenc e imposed a high cost \nbur den on healthcar e sy st ems and pa tien ts, with annual \ntotal medical costs up to nine-times higher for TD pa tien ts \nthan for non-TD pa tien ts ($255,200 vs. $27,800) [ 16 ]. \nOther approaches to managing MF-associated \nanemia include erythropoiesis-stimulating agents, \nc ortic ost er oids, andr ogens such as danazol , \nimmunomodulatory drugs and splenectomy [ 8 , 19 , 20 ]. \nThese strat eg ies have shown modest and transient \nclinical benefit, but none directly target key mechanistic \nc ontribut ors t o MF-associat ed anemia [ 21 , 22 ]. \nMomelotinib is an oral inhibitor that targets JAK1, JAK2 \nand ACVR1 [ 23 , 24 ]. While inhibition of JAK-STAT signaling \nin MF blocks the inflammatory cytokines and clonal pro- \nlifera tion tha t lead to extramedullary hematopoiesis and \nsplenomegaly, ACVR1 inhibition incr eases ir on availabil- \nity, thereby mitigating iron-restricted anemia associated \nwith MF [ 14 , 25 ]. Based on three Phase III trials, momelo- \ntinib impr ov ed splenomegaly, disease-r elated symp- \ntoms and anemia in pa tien ts with JAK inhibitor–naive \nor –experienced MF [ 26–28 ]. In the Phase III MOMEN- \nTUM trial (NCT04173494) of JAK inhibit or–experienc ed, \nsymptoma tic pa tien ts with MF and anemia, momelo- \ntinib demonstrated reduced transfusion burden and \nachieved dur able tr ansfusion independenc e c ompared \nwith danazol, a commonly used trea tmen t specifically for \nMF-associated anemia [ 20 , 28 ]. Given the dual JAK-STAT \nsignaling and ACVR1 inhibitory activities of momelotinib \nand reported clinical trial efficacy, safety and observed \neffects on MF-associated anemia and transfusion depen- \ndence, there is a potential for cost savings and less \nr esour ce utilization with momelotinib r elativ e to other \ntrea tmen ts. The objective of this study was to esti- \nmate the projected differences in total medical costs, \ntransfusion-relat ed c osts and pa tien t time burden associ- \nated with momelotinib vs. danazol from a US perspective. \n2. Materials & methods \n2.1. Data sources \nTo address the study objec tive, projec t ed differenc es \nin cost and time burden associated with momelotinib \nand danazol w er e determined based on transfusion \nindependence data from the MOMENTUM trial and cost \nand time burden data from the published literature. \nMOMENTUM is an in terna tional , double-blind , \nrandomized , contr olled , Phase III study that enrolled \npa tien ts a t 107 sit es across 21 c ountr ies wor ldwide [ 28 ]. \nThe pa tien t popula tion for MOMENTUM w as previously \ndescr ibed [ 28 ]. Br iefly, eligible pa tien ts w er e ≥18 y ears \nwith a confirmed diagnosis of in termedia te-1–, \nin termedia te-2–, or high-r isk pr imar y or secondar y (post- \nPV or post-ET) MF who w er e symptomatic , w er e anemic , \nhad baseline splenomegaly and w er e pr eviously tr eated \nwith an appr ov ed JAK inhibitor. Pa tien ts w er e randomly \nassigned (2:1) to r eceiv e momelotinib (200 mg orally \nonce per day) plus danazol placebo (i.e., the momelotinib \ngroup) or danazol (300 mg orally twice per day) plus \nmomelotinib placebo (i.e., the danazol group) [ 28 ]. Data \non clinical efficacy of momelotinib r elativ e to danazol \nw er e ex trac t ed or calculat ed from the MOMENTUM trial \nfor use in calculations of projected cost and time burden \ndiffer ences betw een these gr oups. \nA target ed lit eratur e sear ch was c onduct ed t o obtain \nUS-based estimates of costs and time burden for pa tien ts \nwith MF. Cost estimates w er e ex trac ted fr om tw o separate \nstudies of pa tien ts with MF, one a study of adults using \nthe IBM M arketS can Commercial Database and the other \na study of adults aged ≥65 years using the Medicare fee- \nfor-service database [ 16 , 17 ]. Overall costs of care were \nex trac ted from the IBM M arketS can Claims study, which \n\nFUTURE ONCOLOGY 2261 \nsummar ized medical, phar macy and transfusion-specific \ncosts of adult pa tien ts with MF [ 16 ]. Cost estima tes \nw er e r eport ed as annual c osts per person in USD, str at - \nified by baseline transfusion status (TD and transfusion \nindependen t/requiring [TI/TR]) ( Supplemen tary Table S1 \nfor definition of transfusion status and Supplementary \nTable S2 for reported costs). Similarly, the Medicare fee- \nfor-service study provided cost- of- care estimates among \npa tien ts with MF aged ≥65 years, stratified by base- \nline transfusion status (TI, TR and TD) ( Supplementary \nTables S1 & S2 ). While diagnoses of iron overload were \ncaptured in the health claims analyses, specific costs \nrelat ed t o iron chelation therapy w er e not available in \nthese data sources. \nTime burden estimates were from a cross-sectional \nw eb-based surv ey fr om the Cooley’s Anemia Foundation \n(the 2022 Knoth et al. study [ 29 ]), which provided \nestimates for a patient’s mean time spent for one RBC \ntransfusion pr ocedur e in pa tien ts with β-thalassemia and \nincluded the following time componen ts: prepara tion, \nw aiting room, w aiting for blood , RBC transfusion pr o- \ncedure and recovery and total round trip travel time \n( Supplementary Table S3 ) [ 29 ]. \n2.2. Analyses of transfusion status & rates in \nMOMENTUM \nData on clinical efficacy of momelotinib r elativ e to dana- \nzol w er e ex trac t ed or calculat ed from the MOMENTUM \ntrial for use in subsequent calculations of projected cost \nand time burden differences between groups. Individual \npa tien t da ta fr om MOMENTUM w er e used t o det ermine \nthe proportion of pa tien ts who were TI, TR and TD \nin the momelotinib and danazol trial arms at baseline \nand at week 24 of the study based on the transfusion \nstatus cr iter ia of the MOMENTUM tr ial ( Supplementary \nTable S1 ) and the average rate of transfusions in each arm. \nThis TI rate at week 24 was based on the prespecified \nterminal 12-week definition of no transfusions and all \nHb levels ≥8 g/dl in the last 12 weeks before week \n24. Analyses w er e r epeated in the subset of pa tien ts \naged 65 years and older. Estimates included only pa tien ts \nwith known transfusion status at week 24 in the base \ncase calculations of project ed c ost and time burden \ndifferenc es. A separat e analy sis also ev alua ted pa tien ts \nbased on a rolling TI rate defined as no transfusions and \nall Hb levels ≥8 g/dl during any 12-week period through \nweek 24 ( Supplementary Table S1 ). Sensitivity analyses \nunder different assumptions about missing transfusion \nstatus w er e also explor ed . All analyses assumed a sus- \ntained effect of trea tmen t on transfusion status. \n2.3. Calculating proje cte d differenc es in c ost & time \nburden \nProject ed medical c osts for momelotinib and danazol, \nstratified by baseline transfusion status, w er e calculated \nby multiplying the cost estimates from the IBM Mar- \nketScan study with the proportion of pa tien ts with TD or \nTI/TR status in each group at week 24 in the MOMENTUM \ntrial . Pr oject ed c ost differenc es w er e calculated as the \ndiffer ence betw een these pr oject ed c osts in the momelo- \ntinib and danazol groups ( Supplementary Figure S1 ). Cost \nestimates for momelotinib and danazol among patients \naged ≥65 years were calculated by multiplying the cost \nestimates from the Medicare study by the proportion \nof pa tien ts aged ≥65 years with TD, TI or TR sta tus \nin each group at week 24 in the MOMENTUM trial, \nand project ed c ost differenc es w er e calculated as the \ndiffer ences betw een these gr oup-specific estimates. The \nproportions of pa tien ts with TD or TI/TR status w er e \nreported in the Clinical Study Report for the overall \npopulation and calculated using individual patient-level \ndata for patients aged ≥65 years. \nProjected outpa tien t transfusion costs for momelo- \ntinib and danazol w er e calculated by multiplying cost \nestimates per outpatient transfusion visit ex trac ted from \nthe literature with rates of transfusion visits for each \ngroup in the MOMENTUM trial. Costs per transfusion \nvisit w er e r eported only in the IBM M arketS can Com- \nmercial Database study; cost estimates from this study \nw er e ther efor e also used in calcula tions for pa tien ts aged \n≥65 years. Rates of transfusion visits for momelotinib \nand danazol pa tien ts w er e r eported in the Clinical Study \nReport for the overall population and calculated using \nindividual pa tien t-level da ta for pa tien ts aged ≥65 years. \nRates of RBC transfusion visits observed over the 24- \nweek trial period in each group were rescaled to reflect \nannualized rates ( Supplementary Figure S1 ). \nPr ojected transfusion-r elated time bur den for \nmomelotinib and danazol was calculated by multiplying \nthe estimated time spent on average per transfusion visit \nfrom the Knoth et al. study [ 29 ] with the average number \nof transfusion visits per pa tien t per year within the \nmomelotinib and danazol arms from the MOMENTUM \ntrial , r espectiv ely. Pr ojected time savings w er e calculated \nas the difference between the projected time burden \nestimates for momelotinib and danazol. ( Supplementary \nFigure S1 ). \n2.4. Sensitivity analysis \nSensitivity analyses w er e performed to assess the robust- \nness of the base case estimates based on two methods \n\n2262 L. MASAROVA ET AL. \nfor imputing unknown transfusion sta tus a t week 24 in \nMOMENTUM. Unknown transfusion status was imputed \neither as TI/TR in both tr ial ar ms or as TD in both trial \narms. These analyses assumed that patients w er e either \nTI/TR vs. TD without the potential for becoming disease- \nfree (i.e., having no trea tmen t c ost). Project ed c ost and \ntime bur den differ ences w er e then r ecalculated under \nthese assumptions. \n3. Results \n3.1. Transfusion status & rates \nA t w eek 24, 143 pa tien ts w er e analyzed (momelotinib \narm, n = 102; danazol arm, n = 41) in the MOMENTUM \ntrial . A t baseline, 45.1% of pa tien ts in the momelotinib \narm w er e TD, and 54.9% of pa tien ts w er e TI/TR; in the \ndanazol arm, 39.0% w er e TD and 61.0% w er e TI/TR \n( Supplementary Table S4A ). Among patients who w er e \nTD at baseline, 60.9% in the momelotinib arm w er e TI/TR \nat the end of the 24-week trial period and 39.1% remained \nTD. In the danazol arm, 37.5% w er e TI/TR and 62.5% \nr emained TD. A t w eek 24, 109 pa tien ts aged ≥65 years \nin the MOMENTUM trial w er e analyzed (momelotinib \narm, n = 76; danazol arm, n = 33) ( Supplementary \nTable S4B ). In this subpopula tion, a t baseline, 47.4% of \npa tien ts in the momelotinib arm w er e TD and 52.6% \nw er e TI/TR; in the danazol arm, 36.4% w er e TD and 63.6% \nw er e TI/TR ( Supplementary Table S4B ). Among patients \naged ≥65 years who w er e TD at baseline, 63.9% in the \nmomelotinib arm w er e TI/TR at the end of the 24-w eek \ntr ial per iod and 36.1% remained TD; in the danazol arm, \n33.3% w er e TI/TR and 66.7% r emained TD. \nPa tien ts in the momelotinib arm had reduc ed rat es of \ntransfusion visits compared with pa tien ts in the danazol \narm among those who w er e TD at baseline (mean, 15.26 \nvs. 26.34 visits/year) and among those who w er e TI/TR at \nbaseline (mean, 5.00 vs. 7.21 visits/year) ( Supplementary \nTable S5A ). Similarly, pa tien ts aged ≥65 years in the \nmomelotinib arm had reduced rates of transfusion visits \ncompared with pa tien ts in the danazol arm among those \nwho w er e TD at baseline (mean, 15.45 vs. 27.34 visits/y ear) \nand those who w er e TI/TR at baseline (mean, 5.81 vs. 6.10 \nvisits/year) ( Supplementary Table S5B ). \n3.2. Proje cte d differences in medical costs \nReduc ed rat es of transfusion dependenc e result ed in \nlow er pr oject ed c osts for momelotinib r elativ e to danazol . \nStratified by TD and TI/TR status at baseline and using \na t erminal TI rat e, the project ed average annual total \nmedical cost savings with momelotinib vs. danazol w er e \n$53,143 per pa tien t with baseline TD status ($116,772 vs. \n$169,915) and $46,455 per pa tien t with baseline TI/TR \nstatus ($35,910 vs. $82,365) ( Figure 1 A). Sensitivity anal- \nyses, assuming pa tien ts with unknown transfusion sta tus \nat week 24 were either all TD or all TI/TR, showed that total \nannual medical service cost savings with momelotinib \nin pa tien ts with baseline TD sta tus ranged from $1,911 \nto $60,938 per person ( Supplementary Table S6 ). Based \non a rolling TI rate, the projected average annual total \nmedical cost savings with momelotinib vs. danazol w er e \n$23,780 per pa tien t with baseline TD status ($204,656 vs. \n$228,437) and $39,305 per pa tien t with baseline TI/TR \nstatus ($105,852 vs. $145,157) ( Supplementary Figure S2 ). \nAmong pa tien ts aged ≥65 years with baseline TD \nstatus, the projected average annual cost of care was \nsimilar in pa tien ts trea ted with momelotinib vs. danazol \n($125,625 vs. $124,618), with a $1,007 savings with \ndanazol ( Figure 1 B). In pa tien ts with baseline TI/TR status, \nthe pr ojected av erage annual cost of car e show ed savings \nof $6,482 per pa tien t - year ($100,296 vs. $106,778) with \nmomelotinib compared with danazol ( Figure 1 B). \n3.3. Proje cte d differences in outpatient transfusion \ncost \nReductions in transfusions w er e associated with savings \nin projected outpa tien t transfusion cost with momelo- \ntinib r elativ e to danazol . Among pa tien ts who w er e TD \nat baseline, there was a projected outpatient transfusion \ncost savings of $42,021 per pa tien t for momelotinib vs. \ndanazol ( Figure 2 A). Among pa tien ts who were TI/TR at \nbaseline, there was a projected outpa tien t transfusion \ncost savings of $8,370 per pa tien t for momelotinib vs. \ndanazol ( Figure 2 A). \nSimilarly, among pa tien ts aged ≥65 years who w er e \nTD at baseline, projected outpatient transfusion cost \nsavings w er e $45,060 per pa tien t with momelotinib vs. \ndanazol ( Figure 2 B). Among pa tien ts aged ≥65 years who \nw er e TI/TR at baseline, pr ojected outpa tien t transfusion \ncost savings w er e $1,107 per pa tien t for momelotinib vs. \ndanazol ( Figure 2 B). \n3.4. Proje cte d differences in transfusion-related \npatient time burden \nReduc ed rat es of transfusions associat ed with momelo- \ntinib also corresponded to reduced transfusion-related \ntime burden for pa tien ts. Among pa tien ts who w er e \nTD at baseline, the pr ojected av erage annual savings \nin transfusion visit time associated with momelotinib \nv s. danazol t otaled 173 h per pa tien t (238 vs. 411 h) \n( Figure 3 A). Projected savings with momelotinib vs. \ndanazol in pa tien ts with baseline TD status included \n68 h for preparation and waiting time, 82 h for trans- \nfusion pr ocedur e and r ecov ery and 24 h for travel time \n( Supplementary Table S7 ). Among patients who w er e \n\nFUTURE ONCOLOGY 2263 \n$116,772\n$35,910\n$169,915\n$82,365\n$0\n$40,000\n$80,000\n$120,000\n$160,000\n$200,000\nBaseline TD Baseline TI/TR\nIBM MarketScan Commercial Database:\nTotal Annual Medical Costs ($USD/Per-Person)†\n-$53,143‡\n-$46,455‡\n$125,625 $100,296$124,618 $106,778\n$0\n$40,000\n$80,000\n$120,000\n$160,000\n$200,000\nBaseline TD Baseline TI/TR\nMomelotinib Danazol Momelotinib Danazol\n$1,007‡\n-$6,482‡\nMedicare Database (patients ≥65 years):\nTotal Annual Medical Costs ($USD/Per-Person)§\nA B\nFigure 1. Projected medical costs based on transfusion status associated with momelotinib vs. danazol. Projected total annual medical \ncosts in $USD/per person based on transfusion status associated with momelotinib vs. danazol based on the IBM MarketScan \nCommercial Database (A) and Medicare database (B) . \n†The IBM MarketScan Commercial Database categorized patients with MF into two groups (TI/TR and TD). Refer to Supplementary \nTable S1. Costs are stratified by transfusion status at baseline. Refer to Supplementary Table S4. Bars r epr esent costs, which w er e \naggr egated acr oss pa tients with known TI/TR or TD sta tus a t week 24. \n‡Differences in cost burden are calculated as cost burden for momelotinib minus cost burden for danazol. Differences < 0 indicate cost \nsavings for momelotinib r elativ e to danazol; differences > 0 indicate cost savings for danazol r elativ e to momelotinib. \n§The Medicare f ee-f or-servic e da tabase ca tegorized pa tien ts with MF in to thr ee gr oups (TI, TR and TD). Refer to Supplementary \nTable S1. Each bar includes costs for patients with a status of TD or TI/TR at week 24; costs are stratified by transfusion status at baseline. \nThe sample size of patients in the MOMENTUM trial who w er e aged ≥65 years was 155. The numbers of individuals in this \nsubpopulation in the momelotinib and danazol arms w er e 101 and 54, r espectiv ely. Refer to Supplementary Table S4. \nMF: My elofibr osis; TD: T ransfusion dependent; TI: T ransfusion independent; TR: T ransfusion requiring; USD: US dollar. \nTI/TR at baseline, the projected average annual savings \nin transfusion visit time associated with momelotinib vs. \ndanazol totaled 34 h per pa tien t (78 vs. 113 h) ( Figure 3 A). \nAmong pa tien ts aged ≥65 years who w er e TD at \nbaseline, pr ojected av erage annual savings in transfusion \nvisit time associated with momelotinib vs. danazol totaled \n186 h per pa tien t (241 vs. 427 h) ( Figur e 3 B). Pr ojected \nsavings with momelotinib vs. danazol in pa tien ts with \nbaseline TD status included 73 h for preparation and \nwaiting time, 88 h for transfusion pr ocedur e and r ecov ery \nand 25 h for travel time ( Supplementary Table S7 ). Among \npa tien ts who w er e TI/TR at baseline, the pr ojected av er- \nage annual savings in transfusion visit time associated \nwith momelotinib vs. danazol totaled 5 h per pa tien t (91 \nvs. 95 h) ( Figure 3 B). \n4. Discussion \nT his analysis sho w ed that r eduction in transfusions asso- \nciated with momelotinib ar e pr oject ed t o result in savings \nin medical and transfusion-related costs and pa tien t time \nbur den r elativ e to danazol in TD and TI/TR pa tien ts with \nMF in the US. \nProject ed c ost and time burden offsets estimat ed \nfor momelotinib w er e larger for TD pa tien ts owing to \nthe larger reductions in transfusions relative to danazol \nin this population compared with the TI/TR patients. \nProjected savings in total annual medical costs with \nmomelotinib compared with danazol were shown using \nboth terminal TI rates and rolling TI rates. A potential \nfor cost savings with momelotinib in TD pa tien ts is \nparticularly relev an t because TD pa tien ts experience \na higher burden than TI/TR pa tien ts. For example, a \nr etr ospectiv e analysis of the Medicare f ee-f or-service \nda tabase estima t ed t otal c osts (medical and pharmacy) \nw er e appr oximat ely 1.8-times g reat er for pa tien ts who \nw er e TD vs. TI and for pa tien ts with sev er e vs. mild \nanemia, demonstra ting tha t achieving or main taining \ntransfusion independence and maintaining Hb levels in \npa tien ts diagnosed with MF can minimize healthcare \nc osts and reduc e the financial burden for pa tien ts and \nhealthcare sy st ems [ 17 ]. Similarly, a r etr ospectiv e analysis \nusing the IBM M arketS can Da tabase estima ted tha t total \nmedical costs w er e appr oximately nine-times higher for \npa tien ts who w er e TD vs. TI [ 16 ]. While w e observ ed some \ndifferences in the magnitude of projected cost and time \nburdens when restricting to pa tien ts aged ≥65 years, \nour findings of potential offsets with momelotinib with \nrespect to transfusion-related costs and time burden were \nc onsist ent with the broader adult popula tion. Insigh ts \non the cost of care for older pa tien ts are particularly \nimportant because MF occurs most frequently in this \npopulation (median age at diagnosis for primary MF, 65–\n70 years), with advanced age correlated with a worse \nprognosis in MF [ 7 , 30 ]. Notably, pa tien ts who require \ntransfusions in the first year of diagnosis w er e older than \npa tien ts who did not r equir e transfusions, with median \n\n2264 L. MASAROVA ET AL. \n$57 ,819\n$18,968\n$99,840\n$27 ,338\n$0\n$20,000\n$40,000\n$60,000\n$80,000\n$100,000\n$120,000\nBaseline TD Baseline TI/TR\nMomelotenib Danazol\n-$42,021\n§\n-$8,370§\nIBM MarketScan Commercial Database: \nTotal Annual Outpatient Visit Costs\n($USD/Per-Person)†,‡\n$58,555\n$22,027\n$103,614\n$23,134\n$0\n$20,000\n$40,000\n$60,000\n$80,000\n$100,000\n$120,000\nBaseline TD Baseline TI/TR\nMomelotinib Danazol\n-$45,060\n§\n-$1,107§\nMedicare Database (patients ≥65 years): \nTotal Annual Outpatient Visit Costs\n($USD/Per-Person)†,‡\nA B\nFigure 2. Projected annual outpatient visit cost per patient based on transfusion status associated with momelotinib vs. danazol. \nProjected annual outpatient visit cost in $USD/per person based on transfusion status associated with momelotinib vs. danazol based \non the IBM MarketScan Commercial Database (A) and Medicare database (B) . \n†Costs of care from the IBM MarketScan Commercial Database and the Medicare f ee-f or-servic e da tabase determined the average c ost \nper person per visit for an outpatient transfusion among those treated for MF. Refer to Supplementary Table S2. Costs are stratified by \ntransfusion status at baseline. Refer to Supplementary Table S4. Bars r epr esent costs, which w er e aggr egated acr oss patients with \nknown TI/TR or TD status at week 24. \n‡The mean number of RBC transfusion visits was calculated using individual patien t -level data from the MOMENTUM trial. RBC \ntransfusions utilized included those r eceiv ed by trial patients and r ecor ded in the GSK raw transfusion data within the 24-week trial \nperiod (inclusive). Under the assumption that an RBC transfusion represented a single transfusion visit, RBC transfusion visits w er e \nstratified by treatment arm and baseline transfusion status such that the mean RBC visits could be calculated for each subgroup. \nDimensional analysis was subsequently used to adjust the time parameter from per 24 weeks to per year. Refer to Supplementary \nTable S5. \n§Differences in cost burden are calculated as cost burden for momelotinib minus cost burden for danazol. Differences < 0 indicate cost \nsavings for momelotinib r elativ e to danazol; differences > 0 indicate cost savings for danazol r elativ e to momelotinib. \nMF: My elofibr osis; RBC: Red blood cell; TD: T ransfusion dependent; TI: T ransfusion independent; TR: T ransfusion requiring; USD: US \ndollar. \nages of 68 (range, 47–85) vs. 57 (range, 28–87) years, \nr espectiv ely [ 31 ]. The differences in the magnitude of \nproject ed c ost and time offset estimat es for the broader \npopulation and the patients aged ≥65 years may be \npartially attribut ed t o differenc es in charact eristics of \npa tien ts in the Medicare f ee-f or-servic e database v s. the \nIBM M arketS can Database. M or e generally, differ ences \nin costs paid by Medicare vs. costs paid by priv a te \ninsur ers captur ed in the M arketS can Database likely \nalso c ontribut e t o the observ ed differ ences, particularly \nwhen considering overall medical costs. Importantly, \nthe pr ojected decr ease in transfusion time burden with \nmomelotinib observed in both the overall population and \npa tien ts aged ≥65 years may c ontribut e t o improving \npa tien ts’ quality of life. In pa tien ts with MF, transfusion \ndependence was shown to have detrimental effects \non multiple aspects of quality of life [ 32 ]. In a post \nhoc analysis of the SIMPLIFY-1 and SIMPLIFY-2 studies, \ng reat er improvement across quality of life domains w er e \nreported in pa tien ts who w er e TD at baseline but \nbecame TI compared with those who remained TD [ 32 ]. \nIn addition, reducing transfusion burden, and thereby \ntra vel time, ma y be especially beneficial for pa tien ts in \nlimited-r esour ce settings including rural areas with fewer \ntransfusion facilities [ 33 ]. \nLimitations of this study include the use of extrap- \nola ted da ta, which pr ovides pr ojections but may not \nreflect the actual cost and time savings with momelotinib \ncompar ed with danazol . An additional limitation may be \nthe differences in the definitions of transfusion status \nbetween data sources. However, for this study, transfusion \ncr iter ia w er e expect ed t o be functionally similar because \nall the studies defined TI status as 0 RBC transfusions \nand TD status as approximately 0.5 transfusions per week. \nFurthermore, an estimate on the costs of transfusion visits \nwas not available in the Medicare study and was approx- \nimated by the estimate from the IBM M arketS can study. \nTher e w er e also differ ences in the pa tien t popula tions and \ntime frame in the MOMENTUM trial compared with the \nIBM M arketS can Database, M edicare database and the \n2022 Knoth et al. study. While MOMENTUM included JAK \n\nFUTURE ONCOLOGY 2265 \n93\n161\n31 44\n112\n194\n37\n53\n33\n56\n11\n15\n0\n50\n100\n150\n200\n250\n300\n350\n400\n450\nMomelotinib Danazol Momelotinib Danazol\nTravel time\nRBCT procedure and recovery\nPreparation and waiting\n§\nBaseline TD Baseline TI/TR\n238\n411\n78\n113\n-173 Hours‡ \n-35 Hours‡ \nIBM MarketScan Commercial Database:\nTime Burden of Transfusion (Hour/Person-Year)†\n95\n167\n36 37\n114\n201\n43 45\n33\n59\n12 13\n0\n50\n100\n150\n200\n250\n300\n350\n400\n450\nMomelotinib Danazol Momelotinib Danazol\nTravel time\nRBCT procedure and recovery\nPreparation and waiting§\nBaseline TD Baseline TI/TR\n241\n427\n91 95\n-186 Hours‡\n-5 Hours‡\nMedicare Database (patients ≥65 years): \nTime Burden of Transfusion (Hour/Person-Year)†,¶\nA\nB\nFigure 3. Pr ojected transfusion-r elated time burden for patients based on transfusion status associated with momelotinib vs. danazol. \nPr ojected transfusion-r elated time burden for patients in h/person-year associated with momelotinib vs. danazol based on the IBM \nMarketScan Commercial Database (A) and Medicare database (B) . \n†Costs of care were extracted from the 2022 Knoth et al. study. This source provided estimates for mean time spent for one RBCT \npr ocedur e stratified by time spent during pr ocedur e pr epar ations, w aiting room, w aiting for blood , RBCT pr ocedur e and r ecov ery and \nr ound-trip trav el time . Refer to Supplementary Table S3. T ime bur dens ar e str atified by tr ansfusion sta tus a t baseline. Refer to \nSupplementary Table S4. Bars r epr esent time burdens, which were aggregated across patients with known TI/TR or TD status at week 24. \n‡Differences in time burden are calculated as time burden for momelotinib minus time burden for danazol. Differences < 0 indicate \ntime savings for momelotinib r elativ e to danazol; differences > 0 indicate time savings for danazol r elativ e to momelotinib. \n§Preparation and waiting included time in preparation, waiting room and waiting for blood. Refer to Supplementary Table S7. \n¶The sample size of patients in the MOMENTUM trial who w er e aged ≥65 years was 155. The numbers of individuals in this \nsubpopulation in the momelotinib and danazol arms w er e 101 and 54, r espectiv ely. Refer to Supplementary Table S4. \nRBCT: Red blood cell transfusion; TD: Transfusion dependent; TI: Transfusion independent; TR: Transfusion requiring; USD: US dollar. \n\n2266 L. MASAROVA ET AL. \ninhibit or–experienc ed, sympt oma tic pa tien ts with ane- \nmia, both the IBM M arketS can and M edicare databases \nincluded both JAK inhibit or–experienc ed and –naive \npa tien ts [ 16 , 17 , 28 ]. Time burden for transfusion was esti- \nmated using the 2022 Knoth et al. study in pa tien ts with \ntransfusion-dependen t β-thalassemia, as estima tes for \npa tien ts with MF w er e not available [ 29 ]. Compared with \nMOMENTUM, this study also had differences in pa tien t \ncharacteristics including age and race [ 29 ]. How ev er, \npa tien ts with β-thalassemia, similar to those with MF, \nhav e r educed RBC pr oduction, which oft en nec essitat es \nRBC transfusions [ 34 ]. Time frames of follow-up also \nvaried across the underlying data sources (i.e., MOMEN- \nTUM assessed transfusion sta tus a t week 24, the IBM \nM arketS can analysis included a 2–calendar year study \nperiod with continuous enrollment for each pa tien t and \nthe 2022 Knoth et al. study reported transfusion time \nbur den ov er a 6-month period); ther efor e, transfusion \nrates, time burden and costs w er e annualized in our \nanalysis [ 16 , 28 , 29 ]. Population sizes also differed and for \npa tien ts aged ≥65 years were especially small in the \ncohort of TD pa tien ts trea ted with danazol (n = 12). \nFinally, early study discon tinua tion in this population \nwith advanc ed, sympt omatic MF and anemia resulted \nin unknown or missing transfusion status at week 24. \nHow ev er, sensitivity analyses w er e performed t o ac c ount \nfor these missing data and showed broadly similar \nresults. Based on these limita tions, in terpreta tion of these \nresults should focus on the overall trend demonstrating \ntransfusion-relat ed c ost and pa tien t time savings with \nmomelotinib compared with danazol rather than the \nnumeric cost values, which may not be generalizable for \nall circumstances. \nAlthough transfusion dependency and anemia are crit- \nical negative prognostic factors in MF and lead to greater \nHCRU, cost and time bur den, tr ea tmen t options for TD \npa tien ts appear to have minimal impact on the under- \nlying cause of MF-associated anemia [ 12 , 16 , 18 , 35 , 36 ]. \nW hile tr ansfusions r apidly incr ease Hb lev els, they do \nnot provide long-term clinical benefits alone and are \nassociated with lower survival , r educed quality of life \nand complications including iron overload [ 8 , 37–39 ]. \nTransfusions also increase the burden on healthcare \nsy st ems because they r equir e infrastructur e, logistics, \nhuman r esour c es and financial capital t o ensure blood \navailability, processing and delivery [ 40 ]. \nMor eov er, transfusion dependence and subsequently \ng reat er HCRU and c osts may r esult fr om my elosuppr es- \nsive JAK inhibitors such as ruxolitinib and fedratinib, \nwhich have been shown to exacerbate anemia [ 41 , 42 ]. \nIn addition, disease-associated or treatment-exac erbat ed \nanemia may lead to JAK inhibitor dose adjustmen ts, in ter- \nruptions or discon tinua tion, which may limit trea tmen t \nefficacy and are associated with poor survival [ 14 , 42–\n46 ]. A r etr ospectiv e r eal-w orld study of 1191 pa tien ts \nwith MF in the US enrolled in the Medicare Adv an tage \nhealth plan r ev ealed tha t significan t HCRU and costs \nremained in pa tien ts trea ted with ruxolitinib, and major \ndrivers of costs included inpa tien t hospitaliza tions and \npharmacy costs [ 47 ]. While this study did not analyze \npa tien ts with TD vs. TI status or identify what proportion \nof costs w er e due to transfusions, mean all-cause and \nMF-relat ed t otal medical c osts w er e incr eased fr om \n6 months before MF diagnosis to 6 months after MF \ndiagnosis (all cause, $24,216 to $48,966 and MF r elated , \n$16,502 to $39,383) [ 47 ]. Beyond JAK inhibitors, common \nsupportiv e tr ea tmen ts for MF-rela ted anemia, including \ndanazol, immunomodulatory drugs and erythropoiesis- \nstimula ting agen ts, have shown inadequa te efficacy, \ndurability and tolerability [ 8 , 19 , 21 , 22 , 35 , 48 , 49 ]. \nIn c ontrast t o these my elosuppr essiv e JAK inhibitors \nand supportiv e tr ea tmen ts, momelotinib decreases aber- \nran t inflamma tion and facilita tes normalized Hb levels, \naddressing a high unmet need in pa tien ts with MF- \nassociated anemia, effects driven at least in part by \nits inhibition of ACVR1 [ 14 ]. In the Phase III MOMEN- \nTUM trial, momelotinib not only demonstrated clinically \nsignificant impr ov ement in MF-associated symptoms, \nanemia measures and spleen response, but also had \nhigher transfusion independence at week 24 compared \nwith danazol (30% vs. 20%; p = .0116, noninferior) [ 28 ]. \nTr ansfusion independence r ate from baseline to week \n24 increased by 17% in the momelotinib group vs. 5% \nin the danazol group [ 28 ]. This analysis using both the \nIBM M arketS can Commercial Database and the Medicare \nf ee-f or-service database demonstrates that reductions in \ntransfusions due to trea tmen t with momelotinib may \ntransla te in to meaningful cost offsets and time savings for \npa tien ts and the healthcare sy st em. \n5. Conclusion \nWhile this study focuses on comparing the projected cost \nand time burden for pa tien ts trea ted with momelotinib \nand danazol in the MOMENTUM trial, additional \nanalyses will assess the economic and humanistic costs \nassociated with momelotinib r elativ e to ruxolitinib \nin JAK inhibitor–naive pa tien ts with MF as well as \nr elativ e to best available therapies in JAK inhibitor \n–experienced pa tien ts using corresponding analyses \nfrom the SIMPLIFY-1 and SIMPLIFY-2 trials. While \nac quisition c osts for momelotinib w er e not y et available \nat the time of the present analysis, budget impact \nand cost effectiveness models that incorporate this \n\nFUTURE ONCOLOGY 2267 \ninforma tion complemen t the r esults described her e \nand may help determine whether reduced transfusion- \nrelat ed c osts and supportiv e car e c osts associat ed \nwith anemia and other comorbidities can offset drug \nc osts [ 50 , 51 ]. A s the present analy sis was limit ed t o the \n24- week r andomized period of MOMENTUM, futur e r eal- \nworld analyses may be required to ev alua te long-term \neffects beyond this time point . A nalyses of r eal-w orld \ndata may also be considered to assess pa tien t costs in \nspecific trea tmen t settings. Given tha t initial regula tory \napproval of momelotinib was in the US, the present \nanalysis is focused on projected cost and time savings \nfrom a US perspective; additional analyses to determine \nimpacts in other markets, which are likely to vary, may be \nw arran ted based on further regulatory decisions. Finally, \nfutur e w ork estimating the time bur den and r esour ce \nutiliza tion associa ted with MF-rela ted transfusions from \nthe provider and pa tien t’s quality of life perspectives will \nbe important to further describe the potential impact of \nmomelotinib. \nArticle highlights \n• Momelotinib is a JAK1, JAK2 and ACVR1 inhibitor that targets both \nthe complex drivers of iron-restricted anemia and chronic \ninflammation in MF, thereby improving constitutional symptoms \nand splenomegaly while also maintaining or improving Hb levels \nacross the continuum of JAK inhibitor–naive and previously JAK \ninhibit or–treat ed patients. \n• Transfusion dependency and moderat e-t o-sev er e anemia are \ncritical negative prognostic factors in MF and lead to significantly \nhigher rates of HCRU and costs in affected patients compared with \nnon-TD patients. \n• MOMENTUM is a randomized, double-blind study comparing \nmomelotinib and danazol in symptomatic patients with MF and \nanemia who previously received JAK inhibitor therapy. \n•P r ojected differ enc es in c ost and time burden associa ted with \nmomelotinib r elativ e to danazol w er e based on patient transfusion \ndata from the MOMENTUM trial and cost estimates from studies \nusing the IBM MarketScan Commercial Database and Medicare \nf ee-f or-servic e da tabase as w ell as time bur den estimates fr om the \n2022 Knoth et al. study. \n• Reductions in transfusions associated with momelotinib are \nproject ed t o r esult in savings in medical and transfusion-r elated \ncosts and patient time savings r elativ e to danazol in patients with \nbaseline TD and TI/TR status, r espectiv ely, using a terminal TI rate: \nannual medical costs ($53,143 and $46,455 per person), outpatient \ntransfusion costs ($42,021 and $8,370 per person) and time \nsavings (173 and 35 h per person). \n•P r o j e c t e d savings in total annual medical costs with momelotinib \nr elativ e to danazol w er e also shown using a rolling TI rate in \npatients with baseline TD and TI/TR status ($23,780 and $39,305). \n•P r o j e c t e d savings in transfusion-related costs and patient time \nsavings with momelotinib r elativ e to danazol w er e also observed \nfor patients aged ≥65 years. \n•T h e results of this analysis demonstrate that momelotinib not only \naddresses a high unmet need in patients with MF-associated \nanemia but may also provide medical cost and time savings for \npatients by reducing their transfusion dependency. \nAcknowledgments \nThis study was funded by GSK. Writing support was provided \nby J Chu (Nucleus Global, an Inizio Company), in ac c ordanc e \nwith the In terna tional Committee of Medical Journal Editors and \nGood Publication Practice guidelines, and was supported by \nGSK. We also thank the pa tien ts and families who participated \nin the trial and all study investigators and site staff. \nAuthor contributions \nAll authors w er e inv olv ed in the c ont ent, writing and revising of \nthis r esear ch article. \nFinancial disclosure \nFunding was provided by GSK. The authors have no other rele- \nv an t affilia tions or financial inv olv emen t with any organiza tion \nor entity with a financial interest in or financial conflict with the \nsubject matter or materials discussed in the manuscript apart \nfrom those disclosed. \nCompeting interests disclosure \nL Masar ova r eports advisory board participation with Cogent, \nGSK, MorphoSys, and PharmaEssentia. S Verst ov sek rec eived \nconsulting fees from GSK. T Liu is a current employee of GSK. \nS Rao is a curr ent employ ee of GSK, has st ock/st ock options \nand r eceiv ed support for attending meetings and/or travel \nfrom GSK. G Sajeev is an employee of Analysis Gr oup, Inc ., \nwhich r eceiv ed consulting fees fr om GSK for this r esear ch. M \nFillbrunn is an employee of Analysis Group, Inc., which received \nconsulting fees from GSK for this r esear ch. R Simpson is an \nemployee of Analysis Group, Inc., which rec eived c onsulting \nf ees from GSK f or this r esear ch. W Li is an employ ee of \nAnalysis Gr oup, Inc ., which r eceiv ed consulting fees fr om GSK \nfor this r esear ch. J Yang is a former employee of GSK. Y Le \nLorier was a former GSK employee. B Gorsh was a former GSK \nemployee and has st ock/st ock options and received support for \nattending meetings and/or trav el fr om GSK and Daiichi-Sankyo. \nJ Sig norovit ch is an employee of Analysis Group, Inc., which \nr eceiv ed consulting fees from GSK for this r esear ch. The authors \nhave no other c ompeting int erests or relev an t affilia tions with \nany organization or entity with the subject matter or materials \ndiscussed in the manuscript apart from those disclosed. \nWriting disclosure \nE ditorial support w as pro vided b y Nucleus Global and funded \nby GSK plc (Philadelphia, PA). Neither GSK plc nor Nucleus Global \ninfluenced the con ten t of this manuscript. \nEthical conduct of r esear ch \nThe MOMENTUM study was c onduct ed in ac c ordanc e with \nthe Declaration of Helsinki and the International Council \nfor Harmonisation guidelines on Good Clinical Practice. The \ninstitutional review board or independent ethics c ommitt ee at \neach study site appr ov ed the pr otocol . A full list of the ethics \n\n2268 L. MASAROVA ET AL. \nc ommitt ees can be found in the associated Supplementary \nMaterial . All participants provided written informed consent. \nData availability statement \nThe authors certify that this manuscript reports the secondary \nanalysis of clinical trial data that have been shared with them, \nand that the use of these shared data is in ac c ordanc e with the \nterms (if any) agreed upon their rec eipt. The sourc e of these data \nis GSK plc (MOMENTUM; ClinicalTrials.gov: NCT04173494). \nPr evious pr esenta tion \nPresented in abstract form at: European Hematology Associa- \ntion 2023 C ongress , June 8–11, 2023, Frankfurt, Germany [ 52 ]. \nORCID \nLucia Masarova \n https://or cid .org/0000-0001-6624-4196 \nReferences \nPapers of special note have been highlighted as: •of interest; ••\nof considerable interest \n1. 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