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HLA-haploidentical Hematopoietic Stem Cell Transplantation in Pediatric Patients With Hemoglobinopathies: Current Practice and New Approaches

HLA-haploidentical Hematopoietic Stem Cell Transplantation in Pediatric Patients With Hemoglobinopathies: Current Practice and New Approaches

Lena Oevermann, Johannes H Schulte, Patrick Hundsdörfer, Dani Hakimeh, Friederike Kogel, Peter Lang, Selim Corbacioglu, Angelika Eggert, Pietro Sodani

Bone Marrow Transplant. 2019 Aug;54(Suppl 2):743-748.

PMID: 31431703

Abstract:

We review current approaches in HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) for pediatric patients with hemoglobinopathies with a focus on recent developments using TCRα/β+/CD19+ depleted grafts in patients with β-thalassemia major (TM) or sickle cell disease (SCD) in two European transplant units. Eleven TM and three SCD patients (Roma cohort) received a preparative regimen consisting of busulfan/thiotepa/cyclophosphamide/ATG preceded by fludarabine/hydroxyurea/azathioprine. The preparative regimen for 5 SCD patients included treosulfan/thiotepa/fludarabine/ATG (Berlin pilot cohort). All grafts were PBSC engineered by TCR-α/β+/CD19+ depletion. In both cohorts, rates for graft failure, treatment related mortality (TRM) and GvHD were encouraging. Overall survival (OS) and disease-free survival (DFS) in the Roma cohort were 84 and 69%, respectively, while OS and DFS are 100% in the Berlin cohort. Immune reconstitution was satisfactory. Although asymptomatic viral reactivation was common, no severe viral infection occured. These data confirm that TCR-α/β+/CD19+ depletion is a well-suited haplo-HSCT strategy for children with hemoglobinopathies. We discuss the results in the context of additional optimization strategies and introduce our concepts for multicenter trial protocols in Germany.

Chemicals Related in the Paper:

Catalog Number	Product Name	Structure	CAS Number	Price
AP299752	Treosulfan		299-75-2	Price

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