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CD34+ Selected Hematopoietic Transplant, an Option When Compliance Is a Concern in Pediatric Patients

Year: 2019
Type: article
Abstract: Background The incidence of non-compliance is as high as 60% in pediatrics, making it a serious concern after hematopoietic stem cell transplant (HSCT), increasing treatment related mortality (TRM), graft versus host disease (GVHD) and decreases overall survival (OS). Risk of GVHD is 30-60%, therefore mitigating this risk when compliance is a concern is crucial. CD34+ selected grafts, have a very low incidence GVHD and can abolish the need for post HSCT immune suppression. Historically T-depleted grafts have been used to reduce incidence of GVHD for patients at risk (e.g. mismatched donor and fanconi anemia (FA) however it has not been studied in the context of compliance. Objectives Assess our institutional experience with pediatric T-depleted HSCT in the setting of risk of non-compliance. We hypothesized that T-depleted grafts result in adequate engraftment without increasing incidence of infections, graft failure and eliminate the risk for GVHD. Methods Retrospective chart review. Patients with FA or a highly mismatched donor were excluded. Results Five pediatric patients' underwent a CD34+ selected HSCT at our institution between 2016 and 2018, using reduced intensity conditioning (alemuzumab, fludarabine, melphalan). Stem cells were collected using the Miltenyi Biotec CliniMACS system. The median age and cell dose at HSCT were 3.4 years and 21.5 × 10 6 TNC/kg respectively. Demographics are summarized in table 1. All 5 patients had aplastic anemia and severe bone marrow failure. Median day of neutrophil and platelet recovery was day +11 and +12 respectively. No post HSCT calcineurin inhibitors were used and there was no GVHD. Morbidity post HSCT was most commonly transient viral reactivation and stool infections. The OS was 100% with sustained engraftment above 95% in 4, and the median follow up of 747 days. One patient had primary graft failure after 3 stem cell infusions and auto recovered at day +46 after last stem cell infusion and demonstrated adequate immune reconstitution after conditioning and HSCT. Conclusion These data support our hypothesis that T-depleted grafts can be an option for patients' with marrow failure syndromes and who are at risk for non-compliance with acceptable morbidity.
Language: English
Subfield: Hematology
Field: Medicine
Open Access status: hybrid
Estimated APC paid: $3,650
OpenAlex ID: W2911503064
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