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T-cell replete haploidentical bone marrow transplantation and post-transplant cyclophosphamide for patients with inborn errors

M. Kurzay,F. Hauck,I. Schmid,V. Wiebking,A. Eichinger,E. Jung,A. Boekstegers,T. Feuchtinger,C. Klein,M. Albert

Published 2019 in Haematologica

ABSTRACT

Matched donor hematopoietic stem cell transplantation (HSCT) cures most patients with inborn hematopoietic disorders, but haploidentical HSCT was historically associated with graft rejection, graft-versus-host disease (GvHD) and infections. In vivo depletion of haploidentical alloreactive T cells with post-transplant cyclophosphamide (haplo pTCy) has been shown to result in outcomes comparable to those with matched related or unrelated donors in adults with malignant diseases. Published results of this approach in patients with nonmalignant diseases are still relatively scarce. We therefore analyzed the results of haplo pTCy in combination with upfront serotherapy and a myeloablative conditioning regimen in patients with non-malignant diseases at our center, showing good overall survival, little GvHD, and few infectious complications. Between 2013 and 2018, all patients with inborn errors of hematopoiesis who lacked a suitable matched related or 10/10 HLA matched unrelated donor, or who had failed a previous HSCT, were treated according to this institutional protocol. The graft was unmanipulated bone marrow from familial HLA-haploidentical donors. Conditioning consisted of alemtuzumab 2x0.2 mg/kg/day (days -9 to -8), fludarabine 5x30 mg/m/d (days -7 to -3), treosulfan 3x14 g/m/d (days -7 to -5), thiotepa 2x5 mg/kg (day -4), and cyclophosphamide 2x14.5 mg/kg/d (days -3 to -2) in all patients except patient n. 5 who received submyeloablative busulfan [total Area Under the Curve (AUC) 55,000 ng x hour/mL] instead of treosulfan/thiotepa with the intention to enable central nervous system engraftment of donorderived microglial cells. GvHD prophylaxis consisted of cyclophosphamide 50 mg/kg/d on days +3 and +4, fol-

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