Outcome of allogeneic haematopoietic stem cell transplantation in myeloproliferative neoplasm, unclassifiable: a retrospective study by the Chronic Malignancies Working Party of the EBMT

Abstract

Summary Myeloproliferative Neoplasm (MPN), unclassifiable (MPN‐U) is a heterogeneous disease with regards to both clinical phenotype and disease course. Patients may initially be asymptomatic or present with leucocytosis or thrombocytosis, anaemia, progressive splenomegaly, constitutional symptom, thromboses or accelerated/blastic phase disease. Treatment strategies are variable and there are no widely accepted consensus management guidelines for MNU‐U. Allogeneic Haematopoietic Cell Transplantation (allo‐HCT) remains the only curative strategy yet outcomes, to date, are not well defined. We hereby report on the largest retrospective study of patients with MPN‐U undergoing allo‐HCT, highlighting the potentially curative role and providing clinicians with robust engraftment, GvHD and outcome data to facilitate patient discussion.

Impact of spleen size and splenectomy on outcomes of allogeneic hematopoietic cell transplantation for myelofibrosis: A retrospective analysis by the chronic malignancies working party on behalf of European society for blood and marrow transplantation (EBMT)

“…This is a registry‐based study conducted on behalf of the Chronic Malignancies Working Party of the European Society for Blood and Marrow Transplantation (EBMT). Information on the process of data registration in EBMT registry, management and extraction has been previously reported 19 . Inclusion criteria were represented by (a) allo‐HCT for chronic phase primary or post‐polycythaemia vera (PPV)/post‐essential thrombocythaemia (PET)‐MF between 2000 to 2017, (b) age ≥18 years, (c) fludarabine‐busulfan (FluBu) or fludarabine‐melphalan (FluMel)‐based conditioning regimens, and (d) availability of splenectomy status at time of allo‐HCT.…”

How I manage myeloproliferative neoplasm‐unclassifiable: Practical approaches for 2022 and beyond

“…Optimal timing of allo-SCT for this disease entity is unknown and clearly, given the incidence, the number of patients requiring, and indeed who are suitable for, this approach remains small. McLornan et al 35 allo-HCT to date. The median age for the myeloablative conditioned cohort (MAC; n = 31) was 52 years and for the reduced-intensity conditioned (RIC) cohort (n = 39) 59 years respectively.…”

“…Optimal timing of allo‐SCT for this disease entity is unknown and clearly, given the incidence, the number of patients requiring, and indeed who are suitable for, this approach remains small. McLornan et al 35 reported on an European Society for Blood and Marrow Transplantation (EBMT) registry‐based cohort of 70 patients with confirmed chronic phase MPN‐U undergoing allo‐HCT from 35 transplant centres, representing the largest report of MPN‐U allo‐HCT to date. The median age for the myeloablative conditioned cohort (MAC; n = 31) was 52 years and for the reduced‐intensity conditioned (RIC) cohort ( n = 39) 59 years respectively.…”

Population Pharmacokinetics of Busulfan and Its Metabolite Sulfolane in Patients with Myelofibrosis Undergoing Hematopoietic Stem Cell Transplantation

“…Genetic aberrations of the genes JAK2, MPL and CALR were identified as the cause of this myeloproliferative disorder [2] and deemed relevant to clinical decision-making with regard to prognosis as well [3]. Since comprehensive mutational profiling has shown that most patients carry a JAKV617F mutation, initial therapy with the JAK1/JAK2 inhibitor ruxolitinib may reduce splenomegaly and improve performance status [4].…”