Allogeneic SCT for Myelofibrosis – pro

Myelofibrosis with myeloid metaplasia (MMM) is a chronic myeloproliferative disorder characterized by bone marrow fibrosis and extramedullary hematopoiesis.
Recently, a retrospective analysis of the outcomes of 320 patients with CIMF receiving allo-SCT between 1989 and 2002 was published. Most patients received ablative conditioning with either total body irradiation (TBI) (n = 117) or busulphan (n = 150) and cyclophosphamide. Bone marrow was the graft source in 208 patients. HLA-identical sibling donors were used in 170 transplants, 117 were from a matched unrelated donor (MUD), and 33 were from an alternative related donor. The 100-day mortality rates were 22% after sibling transplants, 42% after MUD transplants, and 27% after alternative family donor transplants. Corresponding 5-year overall survival rates were 39%, 31%, and 31%, respectively. Multivariate analysis of 215 adult recipients of myeloablative transplants revealed that having an HLA-identical sibling donor, Karnofsky performance score =90%, younger age, more recent date of transplantation, and absence of blasts in peripheral blood prior to transplantation correlated with better survival. Eighteen patients with all of these factors had a 5-year probability of survival of 81%. Although the ideal conditioning regimen is yet to be defined, cyclophosphamide plus busulphan (with busulphan doses adjusted to achieve targeted plasma levels) resulted in better outcomes when compared with TBI-based regimens.
A Tefferi (2016) writes: Considering the lack of effective drug therapy in PMF, the risk of transplant‐related complications might be justified in those patients in whom median survival is expected to be <5 years and leukemic transformation risk >20%. These include DIPSS‐plus high or intermediate‐2 risk patients as well as those with high‐risk mutations
The American Society for Transplantation and Cellular Therapy (ASTCT) considers an allogeneic HSCT “standard of care with clinical evidence” for patients with intermediate and high risk disease.

Kanate AS, Majhail NS, Savani BN, Bredeson C, Champlin RE, Crawford S, et al. Indications for Hematopoietic Cell Transplantation and Immune Effector Cell Therapy: Guidelines from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transplant (2020) 26(7):1247–56

Bacigalupo A, Innocenti I, Rossi E, Sora F, Galli E, Autore F, Metafuni E, Chiusolo P, Giammarco S, Laurenti L, Benintende G, Sica S, De Stefano V. Allogeneic Hemopoietic Stem Cell Transplantation for Myelofibrosis: 2021. Front Immunol. 2021 May 4; 12:637512.

NCCN Myelofibrosis 2022

A. Tefferi, Primary myelofibrosis: 2017 update on diagnosis, risk‐stratification, and management. Hematology Volume91, Issue12 December 2016,
Pages 1262-1271

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