PMS13 Hospital Costs for Xlmtm Patients - a German Perspective

Abstract

X-linked Myotubular Myopathy (XLMTM) is an ultra-rare, life-threatening congenital myopathy characterized by profound muscle weakness, respiratory failure and early death. Due to limited local evidence, we examined inpatient costs, number of admissions and length of stay (LOS) for XLMTM patients admitted to German long-term ventilation (LTV) and acute care (AC) hospitals. Due to XLMTM ultra-rarity, a cross-sectional convenience sample of five XLMTM patients admitted to LTV and AC hospitals was evaluated, including records for three LTV patients and two AC patients admitted between April 2003 and February 2020. Age at admission for two of three LTV patients was >3.5 y.o. and was unknown for the other. AC patients were both < 1 y.o. upon first admission. Hospital costs, admissions, and total LOS were assessed. Costs for patients episodically admitted to AC hospitals were based on diagnosis-related groups whereas costs for LTV patients were based on negotiated per diems. Results were assessed for each patient and stratified by hospital type. The oldest LTV patient drove 71% of the total LOS (8,373 days) and 67% of the total cost (5,072,662€). Due to continuous admission, the LTV patients accounted for 97% of patient days and 90% of total costs. The average LTV per diem was 579.63€ (range 514.00-621.77€). The two patients admitted to ACs in their first year of life generated total hospital costs of 127,847€ and 376,894€ with a calculated average of 1,832€ per diem. The first had six admissions over 227 days with a cumulative LOS of 111 days and the second had 29 admissions over 2,042 days and a cumulative LOS of 150 days. XLMTM drives large clinical and economic burdens on patients affected and the overall German healthcare system. XLMTM patients admitted to LTVs potentially incur higher cumulative costs but lower per diems compared to those admitted to ACs.