How I treat transplant-eligible patients with myelofibrosis

TY - JOUR

T1 - How I treat transplant-eligible patients with myelofibrosis

AU - Kröger, Nicolaus

AU - Wolschke, Christine

AU - Gagelmann, Nico

N1 - © 2023 by The American Society of Hematology.

PY - 2023/11/16

Y1 - 2023/11/16

N2 - Despite the approval of Janus kinase inhibitors and novel agents for patients with myelofibrosis (MF), disease-modifying responses remain limited, and hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment option. The number of HSCTs for MF continues to increase worldwide, but its inherent therapy-related morbidity and mortality limit its use for many patients. Furthermore, patients with MF often present at an older age, with cytopenia, splenomegaly, and severe bone marrow fibrosis, posing challenges in managing them throughout the HSCT procedure. Although implementation of molecular analyses enabled improved understanding of disease mechanisms and subsequently sparked development of novel drugs with promising activity, prospective trials in the HSCT setting are often lacking, making an evidence-based decision process particularly difficult. To illustrate how we approach patients with MF with respect to HSCT, we present 3 different clinical scenarios to capture relevant aspects that influence our decision making regarding indication for, or against, HSCT. We describe how we perform HSCT according to different risk categories and, furthermore, discuss our up-to-date approach to reduce transplant-related complications. Last, we show how to harness graft-versus-MF effects, particularly in the posttransplant period to achieve the best possible outcomes for patients.

AB - Despite the approval of Janus kinase inhibitors and novel agents for patients with myelofibrosis (MF), disease-modifying responses remain limited, and hematopoietic stem cell transplantation (HSCT) remains the only potentially curative treatment option. The number of HSCTs for MF continues to increase worldwide, but its inherent therapy-related morbidity and mortality limit its use for many patients. Furthermore, patients with MF often present at an older age, with cytopenia, splenomegaly, and severe bone marrow fibrosis, posing challenges in managing them throughout the HSCT procedure. Although implementation of molecular analyses enabled improved understanding of disease mechanisms and subsequently sparked development of novel drugs with promising activity, prospective trials in the HSCT setting are often lacking, making an evidence-based decision process particularly difficult. To illustrate how we approach patients with MF with respect to HSCT, we present 3 different clinical scenarios to capture relevant aspects that influence our decision making regarding indication for, or against, HSCT. We describe how we perform HSCT according to different risk categories and, furthermore, discuss our up-to-date approach to reduce transplant-related complications. Last, we show how to harness graft-versus-MF effects, particularly in the posttransplant period to achieve the best possible outcomes for patients.

KW - Humans

KW - Primary Myelofibrosis/drug therapy

KW - Prospective Studies

KW - Hematopoietic Stem Cell Transplantation/methods

KW - Stem Cell Transplantation

KW - Transplantation, Homologous

U2 - 10.1182/blood.2023021218

DO - 10.1182/blood.2023021218

M3 - SCORING: Journal article

C2 - 37647853

VL - 142

SP - 1683

EP - 1696

JO - BLOOD

JF - BLOOD

SN - 0006-4971

IS - 20

ER -