Clinical Complications and Healthcare Resource Utilization Associated with Conventional Management of Sickle Cell Disease with Recurrent Vaso-occlusive Crises and Transfusion-Dependent β-Thalassemia in Germany

Chuka Udeze (), Nanxin Li, Colin Kunzweiler, Jessica Baldwin, Petra Tuzin, Sebastian Dietmar Zingel, Céline Vetter, Silvia Dombrowski, Elena Georgiadou-Schmidt, Aranzazu Alba and Roland Meisel
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Chuka Udeze: Vertex Pharmaceuticals Incorporated
Nanxin Li: Vertex Pharmaceuticals Incorporated
Colin Kunzweiler: Vertex Pharmaceuticals Incorporated
Jessica Baldwin: Vertex Pharmaceuticals Incorporated
Petra Tuzin: Vertex Pharmaceuticals Incorporated
Sebastian Dietmar Zingel: IQVIA Commercial GmbH & Co. OHG
Céline Vetter: IQVIA Commercial GmbH & Co. OHG
Silvia Dombrowski: IQVIA Commercial GmbH & Co. OHG
Elena Georgiadou-Schmidt: Team Gesundheit GmbH
Aranzazu Alba: Team Gesundheit GmbH
Roland Meisel: Heinrich-Heine-University

PharmacoEconomics - Open, 2025, vol. 9, issue 2, No 10, 300 pages

Abstract: Abstract Objective The purpose of this study was to describe clinical complications and healthcare resource utilization (HCRU) among patients with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and patients with transfusion-dependent β-thalassemia (TDT) in Germany. Methods The Betriebskrankenkasse (BKKs) Database was used to identify patients with SCD or TDT. To be eligible for inclusion, patients with SCD were required to have ≥ 2 VOCs/year in any two consecutive years and ≥ 12 months of available data before and after the index date (second VOC in the second consecutive year). Patients with TDT were required to have ≥ 8 red blood cell transfusions (RBCTs) in any 12-month period and ≥ 12 months of available data after the index date (first RBCT). Clinical and HCRU outcomes were analyzed during follow-up. Results Overall, 84 patients with SCD with recurrent VOCs and 68 patients with TDT were identified in the BKKs database. Among patients with SCD with recurrent VOCs, the most prevalent complications were retinopathy (45.2%), multisystem organ disease/failure (40.5%), and mental health complications (31.0%); among patients with TDT, they were endocrine (69.1%) and cardiopulmonary (55.9%) complications and malignancies (44.1%). Patients with SCD experienced a mean of 4.0 (standard deviation [SD] 3.9) VOCs and 1.9 (SD 2.5) hospitalizations per patient per year (PPPY) during follow-up. Patients with TDT had a mean (SD) of 16.4 (11.2) RBCTs and 59.4 (40.8) outpatient visits PPPY. Conclusions Patients with SCD with recurrent VOCs or TDT in Germany experience significant clinical complications and HCRU.

Date: 2025
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DOI: 10.1007/s41669-024-00550-2

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