Haploidentical hematopoietic stem cell transplantation with post‐transplant high‐dose cyclophosphamide in high‐risk children: A single‐center study

Abstract

Background Post‐Cy administration for GVHD prophylaxis in unmanipulated haploidentical HSCT has resulted in improved outcomes in recent years. Studies in children are lacking and accordingly we present the outcomes of 62 haploidentical transplantation for high‐risk children. Procedure We retrospectively assessed 62 transplants in 60 patients who underwent haploidentical‐related HSCT with unmanipulated stem cells and for whom Post‐Cy was used for GVHD prophylaxis. Results Myeloid reconstitution was achieved on day + 30 for 57 of the 62 patients. The median follow‐up of the surviving 39 patients (63%) was 26 months, with a range of 6‐57 months. The OS and EFS at 2 years were 64.6% (52.0%‐77.2%, 95% CI) and 58.9% (46.1%‐71.7%, 95% CI), respectively. The only factor in our multivariate analysis that contributed to an inferior EFS was a poor remission status prior to HSCT (HR, 8.30; 1.08‐63.56; P = 0.041, 95% CI). Conclusion The results of T‐cell replete haploidentical transplantation with Post‐Cy GVHD prophylaxis in high‐risk pediatric patients are promising. However, further research is needed to determine the factors that have affect HLA compatibility for predicting the success of haploidentical transplantations.

T-Cell-Replete Versus ex vivo T-Cell-Depleted Haploidentical Haematopoietic Stem Cell Transplantation in Children With Acute Lymphoblastic Leukaemia and Other Haematological Malignancies

“…Reviewing the literature, however, some trends can be observed (see Table 2 for a summary) (9,12,14,35,41,60,74,75,78,79,95,(99)(100)(101)(102)(103). Generally, the cumulative incidence rates of severe (grade III-IV) aGvHD and overall as well as extensive cGvHD are higher in patients treated with the PTCy platform compared with those treated with ex vivo TCD (Table 1) (35,95).…”

“…In the hHSCT setting using PTCy (113, 61, 75-78, 81, 99, 104, 106, 107, 113), ex vivo α/β TCD (11,36,42,74,102,103), and Beijing protocol (7,16,63,66), no definite conclusions regarding the burden of infectious complications can be concluded using currently published data since not all authors report bacterial, viral and fungal infection prevalence, costs, or prolonged length of stay or mortality due to infectious complications in their cohorts. This may be due to the retrospective nature of most studies.…”

Impact of HLA-DPB1 Matching on Clinical Outcomes after Haploidentical-Related Hematopoietic Stem Cell Transplantation

“…This has stimulated the development of alternative donor sources, including haploidentical grafts, despite this type of haplo-HSCT carries a higher risk for graft failure and GVHD. In this respect, post-transplant Cy administration has resulted in improved outcomes, helping to advance the field of pediatric haplotransplantation 53,54 .…”

Timing of Initiation of Calcineurin Inhibitors in Pediatric Haploidentical Transplantation with Post-Transplantation Cyclophosphamide: Effects on Survival, Relapse, and Cytokine Release Syndrome

“…Infection prophylaxis and microbiological screening were performed as described previously [5]. Neutropenic fever and viremias were treated according to the relevant guidelines, as described previously [5,9]. Cytokine release syndrome Cytokine release syndrome (CRS) was graded according to published criteria [10].…”