Advances in Clinical and Experimental Medicine

Title abbreviation: Adv Clin Exp Med
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ISSN 1899–5276 (print)
ISSN 2451-2680 (online)
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Advances in Clinical and Experimental Medicine

2019, vol. 28, nr 9, September, p. 1223–1228

doi: 10.17219/acem/104688

Publication type: original article

Language: English

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Pediatric unmanipulated haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide and reduced intensity, TBI-free conditioning regimens in salvage transplantations

Elżbieta Wawrzyniak-Dzierżek1,A,B,C,D,E,F, Kornelia Gajek1,B,C,E,F, Aleksandra Ślęzak1,B,C,E,F, Blanka Rybka1,B,C,E,F, Renata Ryczan-Krawczyk1,B,C,E,F, Ewa Gorczyńska1,E,F, Krzysztof Kałwak1,E,F, Marek Ussowicz1,A,B,C,D,E,F

1 Department of Pediatric Bone Marrow Transplantation, Oncology and Hematology, Wroclaw Medical University, Poland


Background. Unmanipulated haploidentical stem cell transplantation (haploSCT) with post-transplant cyclophosphamide is an option for patients with advanced hematologic malignancies. It offers a platform both for non-major histocompatibility complex-restricted alloimmunity due to killer-like immunoglobulin receptor (KIR)-mediated mechanisms of natural killer lymphocyte regulation and for classical T-cell mediated antileukemic effects.
Objectives. The devastating long-term sequelae after total body irradiation (TBI) in children are encouraging omission of irradiation techniques in pediatric stem cell transplantations (SCT).
Material and Methods. Five children, 4 with acute leukemia and 1 with hemophagocytic lymphohistiocytosis, aged from 1 to 10 years, underwent haploSCT with post-transplantation cyclophosphamide. In all children, the conditioning regimen consisted of chemotherapy without TBI. The graft material was bone marrow (BM) in 4 cases and peripheral blood stem cells in 1 case. Three out of 5 leukemic patients showed better KIR haplotype associated with augmented alloreactivity.
Results. Engraftment with complete donor chimerism was achieved in 4 patients, and 1 recipient died before leukocyte recovery. Three patients developed skin acute graft-versus-host-disease (aGvHD), 1 gut aGvHD and 1 liver aGvHD. In 2 recipients, chronic graft-versus-host-disease (cGvHD) was observed (1 limited and 1 extensive). The 4 engrafted patients were alive and in complete remission 3, 9, 32, and 36 months after transplantation. A T-cell count of 200 cells/uL was reached 90 days after haploSCT in all patients.
Conclusion. HaploSCT with TBI-free protocols can be a viable option for heavily pretreated patients with advanced malignancies.

Key words

hematopoietic stem cell transplantation, pediatric, haploidentical, post-transplant cyclophosphamide, TBI-free

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