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Haploidentical stem cell transplantation for congenital bone marrow failure

Severe Aplastic Anaemia Working Party (SAAWP)
Study type:
Study number:
Type of Stem Cell Treatment:
Allogeneic - Haploidentical
Bone marrow failure
Short title:
Haplo-SCT for cBMT
Primary objective:
To assess the outcome of patients affected by congenital BMF after haploidentical HSCT.
Key inclusion criteria:
Inclusion criteria:
• All congenital BMF syndromes before first haploidentical HSCT
• First haploidentical HSCT between 1990-2019
• Stem cell source: bone marrow or peripheral blood (manipulated or unmanipulated)
Principal investigator:
Dr. Stefano Giardino
EBMT Study coordinator:
Brian Piepenbroek
Study coordinator email: