Danazol treatment for poor graft function after hematopoietic stem cell transplantation: a single centre experience

To the Editor, Allogeneic hematopoietic cell transplantation (alloHCT) is the primary treatment option for various malignant and non-malignant hematological disorders. Poor graft function (PGF) is a severe complication of alloHCT, defined by the European Society for Bone Marrow Transplantation (EBMT) as “at least bilinear severe cytopenia and/or transfusion requirement, which occurs in a situation of full donor chimerism” [1]. PGF has a complex pathophysiology, involving both intrinsic hematopoietic stem cell and bone marrow microenvironment factors. Extra-medullary elements, such as GvHD, donor-specific antibodies, and viral reactivation, also contribute to this scenario, causing immune imbalance and dysregulation of stem cell function [2]. The reported incidence of PGF ranges from 5 to 27% [3, 4], depending on the definition of PGF used, and varies according to donor type, conditioning regimen, patient disease, CMV serological status, CD34+ dose, and graft source. PGF contributes to transplant-related morbidity and mortality, increasing patients’ susceptibility to infectious and hemorrhagic complications, as well as transfusion burden and in-hospital stay. This is a preview of subscription content, access via your institution Access options Subscribe to this journal Receive 12 print issues and online access 251,40 € per year only 20,95 € per issue Buy this article - Purchase on SpringerLink - Instant access to the full article PDF. 39,95 € Prices may be subject to local taxes which are calculated during checkout References Sureda A, Corbacioglu S, Greco R, Kroger N, Carreras E. The EBMT Handbook. Cham: Springer International Publishing; 2024. Man Y, Lu Z, Yao X, Gong Y, Yang T, Wang Y. Recent advancements in poor graft function following hematopoietic stem cell transplantation. Front Immunol. 2022;13:911174. Olsson R, Remberger M, Schaffer M, Berggren DM, Svahn BM, Mattsson J, et al. Graft failure in the modern era of allogeneic hematopoietic SCT. Bone Marrow Transpl. 2013;48:537–43. Lee K-H, Lee J-H, Choi S-J, Lee JH, Kim S, Seol M, et al. Failure of trilineage blood cell reconstitution after initial neutrophil engraftment in patients undergoing allogeneic hematopoietic cell transplantation – frequency and outcomes. Bone Marrow Transpl. 2004;33:729–34. Scheckenbach K, Morgan M, Filger-Brillinger J, Sandmann M, Strimling B, Scheurlen W, et al. Treatment of the bone marrow failure in Fanconi anemia patients with danazol. Blood Cells Mol Dis. 2012;48:128–31. Shah S, Yadav R, Bhattarai A, Tharu S, Sharma P, Subedi P, et al. Danazol for the treatment of myelodysplastic syndromes: a systematic review. Oncology (Williston Park). 2023;37:480–7. Cervantes F, Isola IM, Alvarez-Larrán A, Hernández-Boluda J-C, Correa J-G, Pereira A. Danazol therapy for the anemia of myelofibrosis: assessment of efficacy with current criteria of response and long-term results. Ann Hematol. 2015;94:1791–6. Shah S, Yadav R, Bhattarai, Dahal K, Tharu S, Gautam S, et al. Safety and efficacy of danazol in immune thrombocytopenia: a systematic review. Res Pr Thromb Haemost. 2024;8:102444. Gardner FH, Juneja HS. Androstane therapy to treat aplastic anaemia in adults: an uncontrolled pilot study. Br J Haematol. 1987;65:295–300. Sanchez-Medal L, Gomez-Leal A, Duarte L, Rico MG. Anabolic androgenic steroids in the treatment of acquired aplastic anemia. Blood. 1969;34:283–300. Townsley DM, Dumitriu B, Liu D, Biancotto A, Weinstein B, Chen C, et al. Danazol treatment for telomere diseases. N Engl J Med. 2016;374:1922–31. Calado RT, Yewdell WT, Wilkerson KL, Regal JA, Kajigaya S, Stratakis CA, et al. Sex hormones, acting on the TERT gene, increase telomerase activity in human primary hematopoietic cells. Blood. 2009;114:2236–43. Bar C, Huber N, Beier F, Blasco MA. Therapeutic effect of androgen therapy in a mouse model of aplastic anemia produced by short telomeres. Haematologica. 2015;100:1267–74. Author information Authors and Affiliations Contributions FS, NG, and GS designed the study. FS, NG, and FC collected data. FS and NG performed data analysis and wrote the manuscript. FS designed table and figures. KB, NC, MG, and FP were involved in results discussion and helped writing the manuscript. All authors approved the final version of the manuscript. Corresponding author Ethics declarations Competing interests The authors declare no competing interests. Additional information Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations. Rights and permissions About this article Cite this article Serpenti, F., Galli, N., Cavallaro, F. et al. Danazol treatment for poor graft function after hematopoietic stem cell transplantation: a single centre experience. Bone Marrow Transplant 61, 360–363 (2026). https://doi.org/10.1038/s41409-025-02790-0 Received: Revised: Accepted: Published: Version of record: Issue date: DOI: https://doi.org/10.1038/s41409-025-02790-0