Costs of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) in Pediatric Patients with Acute Lymphoblastic Leukemia (ALL) - an Analysis of German Claims Data
Authors: Lehne Moritz , Hickstein Lennart , Salimullah Tayyab , Su Jun , Prieur Sigurd , Thomas Simu K 
Published: November 2019

Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is a possible treatment option for pediatric patients with acute lymphoblastic leukemia (ALL). Typical patient groups eligible for HSCT are high-risk patients, refractory patients or patients with a relapse after initial chemotherapy treatment. Despite constituting a potentially curative treatment for pediatric ALL patients, HSCT is a complicated procedure that requires extensive medical care and can result in life-threatening complications. Furthermore, HSCTs are associated with significant costs, not only related to the procedure itself but also to follow-up medical care. The aim of this study is to provide estimates of health care expenditures for pediatric patients with ALL undergoing allogeneic HSCT in Germany. Costs are considered from the perspective of the German statutory health insurance (i.e. from a payers' perspective).
In a retrospective observational study based on German claims data, health care expenses for allogeneic HSCTs in pediatric ALL patients were investigated. As a data source, we used the German Health Risk Institute (HRI) database - a representative database including approximately 7 million patient journeys over a continuous observation period of 6 years. The study population comprised pediatric patients (< 18 years) with an HSCT procedure between January 1, 2010 and June 30, 2014 and a diagnosis of ALL (ICD-10 code: C91.0*) in the same hospital case as the HSCT. Costs were analyzed for a time interval reaching from 2 quarters prior to the HSCT to 8 quarters after the HSCT (the analysis was done in quarters because outpatient diagnoses in German claims data are documented on a quarterly basis). The following health care costs were considered in the analysis: (1) costs for prescription medication, (2) outpatient treatment costs, (3) inpatient treatment costs, (4) costs for remedies and technical aids (e.g. physiological or occupational therapy). Other expenses such as costs for over-the-counter medication, patient transport or costs associated with the stem cell donor were not included in the analysis. In addition, we analyzed the health resource utilization of patients (hospitalization days, outpatient visits).
A total of 1,687 individuals with an HSCT procedure in the identification period (January 1, 2010 to June 30, 2014) were identified. Of these, 30 pediatric patients (12 female) with a diagnosis of ALL were included in the final study population. Mean age at the time of the HSCT was 9.3 years (SD: 5.3). Mortality in the 4 (8) quarters following the HSCT procedure was 10.0 % (13.3 %). The average number of hospitalization days per patient in the time period from 2 quarters before the HSCT to 8 quarters after the HSCT was 291.5 (SD: 166.78) with an average of 45.5 days (SD: 42.4) for emergency hospitalizations. A mean of 43.2 outpatient visits (SD: 22.1) was observed in this time period.
Average total costs per patient from 2 quarters before the HSCT to 8 quarters after the HSCT were € 290,125 (SD: 110,381) ranging from € 149,933 to € 597,774 €. The largest proportion of these costs were incurred in the hospital sector, followed by costs for medication and ambulatory treatment (see Table 1). Total costs per patient were highest in the index quarter with the HSCT procedure (€ 133,585). However, substantial costs were also observed in the quarters before and after the HSCT with costs gradually decreasing in the quarters following the HSCT procedure (see Figure 1).
Our results provide first estimates for HSCT treatment costs in pediatric ALL patients for different health care sectors from the perspective of the German statutory health insurance.
Table 1 Costs per patient (in €) in the observation period (two quarters before the HSCT to eight quarters after the HSCT). Table 1. Costs per patient (in €) in the observation period (two quarters before the HSCT to eight quarters after the HSCT). Figure 1 Total costs per quarter of observation period (QO: index quarter with HSCT procedure). Figure 1. Total costs per quarter of observation period (QO: index quarter with HSCT procedure).

Disclosures
Lehne: Elsevier: Employment. Hickstein:Elsevier: Employment; Health Risk Institute: Employment. Salimullah:Novartis Pharma AG: Employment. Su:Biogen: Employment, Equity Ownership; Novartis Pharmaceuticals Corporation: Employment. Prieur:Elsevier: Employment. Thomas:Novartis Pharmaceuticals Corporation: Employment.