Allogeneic peripheral blood stem cell transplantation (PBSCT) from HLA-identical sibling donors in children with hematological diseases: a single center pilot study

Abstract

Summary:Between February 1995 and July 1999 25 pediatric patients (8 months to 14 years old) underwent peripheral blood stem cell transplantation (PBSCT) from an HLA-identical sibling donor. Diagnoses included ALL (17), non-ALL (6), and non-malignant disease (2). GVHD prophylaxis consisted of cyclosporine plus methotrexate (15), only cyclosporine (8), cyclosporine plus prednisone (1), or nothing (1). All donors (6 months to 41 years old) received G-CSF at 10 g/kg/day subcutaneously for 4-5 days and on day 5 underwent large volume leukapheresis. Median number of CD34 + and CD3 + cells collected and infused was 6.9 × 10 6 (range 2.5-32.8) and 4.5 × 10 8 (0.5-22.1) per kg of recipient body weight respectively. Median time to achieve ANC Ͼ0.5 × 10 9 /l and platelets Ͼ20 × 10 9 /l was 10 and 12 days, respectively. Acute GVHD grade уII developed in 10 of 24 evaluable patients (42%). Probability of acute GVHD was 62%. Median time to discharge was 25 days (range 14-52). Among 20 evaluable patients, five (25%) developed chronic GVHD at day 100. Probability of chronic GVHD was 29% after 1 year post PBSC. At a median follow-up of 558 (9-2071) days, overall survival for the whole group is 68%. Probabilities of event-free survival, overall survival and relapse for patients with malignant hematological diseases are 53%, 59% and 24% at 5 years, respectively. This study has confirmed the feasibility and safety of mobilization and collection of PBSC products and the applicability of this procedure to the pediatric population, both donors and recipients. Studies including larger numbers of pediatric patients undergoing allogeneic PBSCT are warranted to determine the long-term outcomes of such procedures. Bone Marrow Transplantation (2001) 28, 537-543.

Non-TBI Hematopoietic Stem Cell Transplantation in Pediatric AML Patients

“…While some studies report a higher incidence of chronic GVHD with PBSC transplantation, 38-41 others do not indicate a difference in chronic GVHD. 42 Our results demonstrate that although the difference is not statistically significant, the use of allogeneic PBSCs rather than BMSCs may lead to better OS in recipients of matched sibling allografts for pediatric AML patients.…”

“…36 Although several studies document more rapid hematopoietic recovery after PBSC transplantation in adult and pediatric patients, [37][38][39][40][41][42] there is still substantial controversy regarding acute and chronic GVHD, TRM, relapse, and survival. While some studies report a higher incidence of chronic GVHD with PBSC transplantation, 38-41 others do not indicate a difference in chronic GVHD.…”

Chronic Graft-Versus-Host Disease (GVHD) in Children

“…Patients in this study had similar rates of aGVHD, but rates of cGVHD were higher with a relative risk of 1.85 in the PBSC group [17]. Thus, despite the lack of randomized studies in children it appears that he risk of cGVHD with PBSC is higher than BM, though it is unclear of the overall effects on TRM and relapse free survival [96,97]. …”

Peripheral blood progenitor cell collection adverse events for childhood allogeneic donors: variables related to the collection and safety profile

“…According to the European Bone Marrow Transplantation (EBMT) data, it is estimated that about 500–600 children become donors of haematopoietic progenitor cells (HPC) for siblings undergoing allogeneic stem cell transplantation in Europe every year (Stycznski, personal communication). Although most institutions used bone marrow HPC, several studies have reported on the use of children as donors for peripheral blood progenitor cells (PBPC) (Kawano et al , 1999; Levine et al , 2000; Benito et al , 2001; González‐Vicent et al , 2002; Watanabe et al , 2002; Pérez et al , 2007). However, there is little information regarding donor safety and early side effects of PBPC collection, and variables predicting the yield.…”