Allogeneic Stem Cell Transplantation for Children with Sickle Cell Disease Achieves Quality of Life Similar to Normal Children and Is Cost Effective

Allogeneic stem cell transplantation (alloSCT) remains the only curative option for sickle cell disease (SCD).  However, no systematic analysis exists comparing cost and quality of life (QOL) among this population. We investigated the QOL outcomes and health care utilization associated with alloSCT in children with SCD.

Internal financial data from 2002-2011 was analyzed retrospectively across two groups – post-alloSCT patients (>day+365) and patients with SCD referred for alloSCT and/or HLA typed. Surviving alloSCT recipients (A) and SCD controls (B) were surveyed with age appropriate Pediatric Quality of Life Inventory (PedsQLª) and EuroQOL (EQ-5Dª) questionnaires. Group A siblings without SCD (C) were also surveyed as unaffected controls. Mean QOL scores were calculated for each group with a max score of 100. Utility scores were determined based on EQ-5D responses. These scores and costs for groups A and B were used to calculate cost per quality adjusted life month (QALM) for the cohort of patients surveyed. Wilcoxon test was used to determine statistical significance.

Group A, B, and C had 16 (mean age - 14yrs), 19 (mean age - 12yrs), and 14 children (mean age - 14yrs), respectively. SCD therapy included hydroxyurea (group A n=8, group B n=10) and chronic transfusions (group A n=7, group B n=2). Mean PedsQL scaled scores were 83, 81, and 88, respectively. Mean EQ-5D visual analogue scale scores were 92, 87, 96, respectively. Mean utility scores were 0.87, 0.91, and 0.89, respectively.  All QOL scores were not statistically significant (p = 0.2638). Healthcare utilization among groups A and B was previously reported (see details in table below). The median inpatient cost per QALM for group A was $0 and $514 for group B (p = 0.0023).  Outpatient cost per QALM for group A was $353 and $236 for group B (p = 0.3506). 

SCD patients' post-alloSCT QOL scores are similar to unaffected siblings, indicating that QOL has normalized. Controls with SCD also had scores similar to unaffected controls. However, a statistically significant difference exists in the inpatient cost per QALM post-alloSCT compared to controls with SCD. Outpatient was not significant which may reflect the limitations of the study period as post-alloSCT QOL and cost can change over time (Felder-Puig 2006; Majhail 2010). Ultimately, this study provides the first combined analysis of QOL as an outcome and the economic impact of alloSCT for pediatric SCD patients. Further analysis is ongoing to affirm that alloSCT is a beneficial and cost effective management option for patients.

	Post-AlloSCT (>D+365, group A)	Controls (group B)	p-value
Mean outpatient visits	0.93	1.81	<0.0001
Mean outpatient cost ($)	831.10	739.2	0.840
Mean outpatient cost per QOL ($/QALM)	723.67	442.16	0.3506
Mean inpatient visits	0.02	0.52	<0.0001
Length of stay	0.06	1.05	<0.0001
Mean inpatient cost ($)	293.80	2050.20	0.115
Mean inpatient cost per QOL ($/QALM)	345.69	769.08	0.0023