Thiotepa–busulfan–fludarabine (TBF) conditioning regimen in patients undergoing allogeneic hematopoietic cell transplantation for myelofibrosis: an outcome analysis from the Chronic Malignancies Working Party of the EBMT

• Published in: Bone marrow transplantation (Basingstoke) Vol. 56; no. 7; pp. 1593 - 1602
• Main Authors: Battipaglia, Giorgia, Mauff, Katya, Wendel, Lotus, Angelucci, Emanuele, Mohty, Mohamad, Arcese, William, Santarone, Stella, Rubio, Marie Therese, Kroger, Nicolaus, Fox, Maria Laura, Blaise, Didier, Iori, Anna Paola, Fanin, Renato, Chalandon, Yves, Pioltelli, Pietro, Marotta, Giuseppe, Chiusolo, Patrizia, Sever, Matjaz, Solano, Carlos, Contentin, Nathalie, de Wreede, Liesbeth C., Czerw, Tomasz, Hernandez-Boluda, Juan Carlos, Hayden, Patrick, McLornan, Donal, Yakoub-Agha, Ibrahim
• Format: Journal Article
• Language: English
• Published: London Nature Publishing Group UK 01.07.2021; Nature Publishing Group
• Subjects: 692/699/1541/1990/2331; 692/699/67/1990/2331; Age; Antilymphocyte serum; Bone marrow; Busulfan; Care and treatment; Cell Biology; Conditioning; Donors; Dosage and administration; Drug therapy, Combination; Fludarabine; Globulins; Graft versus host disease; Graft vs Host Disease; Graft-versus-host reaction; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Histocompatibility antigen HLA; Human health and pathology; Humans; Immunology; Immunotherapy; Internal Medicine; Leukocytes (neutrophilic); Life Sciences; Medical prognosis; Medicine; Medicine & Public Health; Middle Aged; Multivariate analysis; Myelofibrosis; Neoplasms; Neutrophils; Patient outcomes; Patients; Primary Myelofibrosis; Public Health; Retrospective Studies; Stem cell transplantation; Stem Cells; Thiotepa; Thymocytes; Transplantation; Transplantation Conditioning; Transplants & implants; Variables; Vidarabine - analogs & derivatives; Vidarabine - therapeutic use; Europe
• ISSN: 0268-3369; 1476-5365
• DOI: 10.1038/s41409-021-01222-z

Allogeneic hematopoietic cell transplantation (allo-HCT) remains the only curative option in MF. There is no consensus on the optimal conditioning regimen. We report outcomes of 187 patients with MF transplanted between 2010 and 2017 conditioned with TBF. Median age was 58 years. Median interval from diagnosis to allo-HCT was 44 months. Donors were haploidentical (41%), unrelated (36%) or HLA-identical siblings (23%). Stem cell source was PB in 60%. Conditioning was myeloablative in 48% of cases. Antithymocyte globulin (ATG) was used in 41% of patients. At 100 days, neutrophil and platelet engraftment were 91% and 63% after a median of 21 and 34 days, respectively. Grade II-IV and III-IV acute GVHD occurred in 24% and 12%, while at 3 years, all grade chronic GVHD and chronic extensive GVHD had been diagnosed in 38% and 11%. At 3 years, OS, RFS and GRFS were 55%, 49% and 43%, respectively. RI and NRM were 17% and 33%. On multivariate analysis, poor KPS and the use of unrelated donors were associated with worse GRFS and a higher grade II-IV acute GVHD, respectively. Neither donor type nor intensity of the conditioning regimen influenced survival outcomes. TBF is a feasible conditioning regimen in allo-HCT for MF in all donor settings although longer term outcomes are required.