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Allogeneic hematopoietic stem cell transplantation for the BCR-ABL1 negative myeloproliferative neoplasms in children

Paediatric Diseases Working Party (PDWP)
Study number:
Short title:
Allo-HSCT for Ph-neg MPN in children
Primary objective:
To evaluate in children transplanted with allogeneic HSC for Ph-neg MPNs:
a) cumulative incidence of relapse (CIR) ;
b) non-relapse mortality (NRM);
d) leukemia-free survival (LFS);
e) event-free survival (EFS);
Key inclusion criteria:
a. Children and adolescents with the classical Ph-neg MPNs (polycytemia vera, PV; essential thrombocythemia, ET; primary myelofibrosis, PMF; post-PV PMF (post-polycythemia (PPV) myelofibrosis (MF)); post-ET PMF( post-essential thrombocythemia (PET) myelofibrosis (MF)), Ph-neg MPN transformed to MDS or acute leukemia);
b. Matched sibling and related or unrelated matched or mismatched donor;
c. Hematopoietic stem cell source - bone marrow, peripheral blood or cord blood;
d. Age ≤ 18 years;
e. Analyzed time period: January 1st 2000 – December 31st 2016.
Principal investigator:
Jacek Wachowiak
EBMT Study coordinator:
Arnaud Dalissier
Study coordinator email: