Risk Factors for Transplant Outcomes in Children and Adolescents with Non-Malignant Diseases Following Allogeneic Hematopoietic Stem Cell Transplantation

Abstract

Background The objective of this study was the analysis of transplant outcomes and survival in children treated with allogeneic hematopoietic cell transplantation (alloHCT) for non-malignant disorders, with a focus on risk factor analysis of transplant-related mortality (TRM). Material/Methods The treatment outcome was analyzed retrospectively in 10 consecutive years in 4 pediatric transplant centers in Poland. To compare the outcomes, patient data were analyzed according to the diagnosis, age at transplant, donor type, stem cell source, conditioning regimens, transplanted CD34+ cells dose, and pediatric TRM score. Results From 183 analyzed patients, 27 (14.8%) died, all of them due to transplant-related complications. TRM occurred more frequently in matched unrelated donor (MUD) transplant recipients vs. matched sibling donor (MSD) transplant recipients (p=0.02); in peripheral blood (PB) recipients vs. bone marrow (BM) recipients (p=0.004); and in patients receiving >5×10 6 /kg CD34+ cells (p<0.0001). OS differed significantly according to underlying disease comparing to other diagnoses. Lower survival was found in patients transplanted from MUD (p=0.02). OS was higher in patients receiving BM (p=0.001) and in those receiving ≤5×10 6 /kg CD34+ cells (p<0.001) . Multivariate analysis showed lower probability of TRM in BM recipients (p=0.04). The probability of TRM was higher in SCID patients (p=0.02) and in patients receiving >5×10 6 /kg CD34+ cells (p=0.0001). Conclusions Underlying disease, stem cell source, and CD34+ dose higher than 5×10 6 /kg were the most important risk factors for TRM, and they all affected OS.

The impact of donor total estimated blood volume on nucleated cell yield in bone marrow harvests for hematopoietic stem cell transplantation

“…Optimal counts appear to occur at 4 × 10 8 /kg, with even higher counts showing an association with increased mortality. [19][20][21] We were able to provide transplant centers with an average of more than 4 TNC × 10 8 / kg of nucleated cells, missing the target of 2 × 10 8 /kg in 1.7% of cases.…”

Total nucleated cell dose in graft is a better prognostic factor for survival in pediatric patients transplanted with bone marrow compared to CD34+, CD3+, or total mononuclear cell count

“…There are many studies dealing with the relationship between some transplant-related parameters and the success of allogeneic HSCT in literature. [1][2][3][4][5][6][8][9][10] In the pediatric age-group, however, there are only a few studies dealing with the number of cells in grafts and transplant outcome. 3,4,8 Published studies investigating the outcome of cell dose parameters in allogeneic transplant, with either matched sibling donors or with matched unrelated donors, and with either bone marrow or peripheral blood as stem cell source, have yielded rather inconsistent results.…”

“…[1][2][3][4][5][6][8][9][10] In the pediatric age-group, however, there are only a few studies dealing with the number of cells in grafts and transplant outcome. 3,4,8 Published studies investigating the outcome of cell dose parameters in allogeneic transplant, with either matched sibling donors or with matched unrelated donors, and with either bone marrow or peripheral blood as stem cell source, have yielded rather inconsistent results. We could find only a limited number of Englishlanguage studies including analyses of different productrelated parameters on survival and transplant-related outcomes in children.…”

“…There are many studies dealing with the relationship between some transplant‐related parameters and the success of allogeneic HSCT in literature 1‐6,8‐10 . In the pediatric age‐group, however, there are only a few studies dealing with the number of cells in grafts and transplant outcome 3,4,8 .…”

Persistent Musculoskeletal Deficits in Pediatric, Adolescent and Young Adult Survivors of Allogeneic Hematopoietic Stem-Cell Transplantation

“…(2)(3)(4) However, the treatment efficacy of alloHSCT must be balanced by the risk of adverse outcomes that are associated with diminished quality of life and increased mortality risk. (5)(6)(7)(8) AlloHSCT regimens, which include chemotherapy, total body irradiation (TBI), and immune suppressive therapies, and their ensuing complications pose numerous risk factors for bone accrual and bone health including malnutrition, vitamin D deficiency, and reduced muscle strength. (8)(9)(10) Graft-versushost-disease (GVHD) and dysregulation of the immune system further impose threats to skeletal recovery through osteoclast activation and diminished osteoblast function.…”