Allogeneic Hematopoietic Cell Transplantation for Children with Sickle Cell Disease Is Beneficial and Cost-Effective: A Single-Center Analysis

Abstract

Limited data exist regarding health care utilization (HCU) in patients receiving allogeneic hematopoietic cell transplantation (alloHCT) for sickle cell disease. Financial data from 2002 to 2011 were analyzed for 26 alloHCT patients and 48 control subjects (referred but without alloHCT). HCU of alloHCT was determined over 3 time periods: pre-alloHCT, during alloHCT (day 0 to day +365), and post-alloHCT. The median total cost per patient during the alloHCT year was $413,000 inpatient and $18,000 outpatient. Post-alloHCT HCU decreased when compared with pre-alloHCT and control subjects. The median cost of post-alloHCT outpatient visits per patient was significantly less when compared with pre-alloHCT (P = .044). The median cost of post-alloHCT inpatient visits per patient approached significance when compared with those pre-alloHCT (P = .079). Sixteen post-alloHCT patients, 19 control subjects, and 14 unaffected siblings were surveyed using Pediatric Quality of Life Inventory and EuroQOL questionnaires; however, the questionnaire scores across all 3 patient groups were not statistically significant (P = .2638). When adjusted for health-related quality of life, the analysis suggested alloHCT has a positive impact on health-related quality of life over control subjects. These pilot data support our hypothesis that alloHCT in children with sickle cell disease reduces HCU compared with control subjects without alloHCT.

Improved Splenic Function After Hematopoietic Stem Cell Transplant for Sickle Cell Disease

“…Finally, while the aim of this study was not to describe event‐free survival outcomes for patients who received HSCT for SCD, it is worth noting that 70/71 (98.6%) of patients transplanted at the two study institutions with an HLA‐identical sibling donor were alive and without SCD at the time of last clinical follow‐up. This observation and our primary findings that HSCT improves splenic function in most patients further support the growing literature documenting the benefits of HLA‐identical sibling donor HSCT for patients with SCD . Even with an improvement in splenic function, patients cured of SCD after HSCT may still face an increased risk for invasive bacterial infections.…”

Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation

“…Five-year survival is excellent, considering that these transplants were performed worldwide and reported to observational registries and that the majority of patients were not enrolled on clinical trials. [22][23][24][25][26][27] Further, we were able The adjusted Cox regression analysis was stratified by registry (EBMT and CIBMTR); age was considered as a continuous variable, and when considering the graft source, PB and CB were compared, separately, with BM (baseline) for the EFS.…”

National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: The Healthcare Delivery Working Group Report

“…Successful HCT may decrease health care utilization, as has been reported in patients with sickle cell disease, as compared to controls who did not undergo transplantation. 12 While quality of care is the fundamental goal of long-term management of patients, value of care is equally important for sustainability of the health care system. “Value” which in simple terms is the ratio of quality over cost, can integrate other healthcare goals such as safety, patient-centeredness, efficiency, cost containment, equity and access.…”