CLINICAL AND ECONOMIC BURDEN OF TRANSFUSION-DEPENDENT BETA-THALASSEMIA IN FRANCE- A RETROSPECTIVE ANALYSIS OF THE FRENCH NATIONAL HEALTH DATA SYSTEM (SNDS)
Author(s)
Brousse V1, Badens C2, Quignot N3, Qi L4, Gatta F5, Paramore C6, Undreiner L7
1Hôpital Universitaire Necker Enfants Malades, Paris, France, 2Aix Marseille University, Marseille, France, 3Certara, Paris, 75, France, 4Certara, Nantes, France, 5bluebird bio (Switzerland) GmbH, Zug, Switzerland, 6bluebird bio Inc, Cambridge, MA, USA, 7bluebird bio France, Paris, France
OBJECTIVES: Transfusion-dependent β-thalassemia (TDT) is a rare genetic hemoglobinopathy with currently limited curative options. This study aims to describe the burden of TDT in France. METHODS: Retrospective analysis of the SNDS database, abstracting data from 01-01-2007 to 31-12-2016. Patient identification criteria included: ≥1 occurrence before age 50 of one of the ICD10 β thalassemia-related codes (D56.1, D56.2 D56.8 or D56.9) in hospitalization records and in Long Term Sickness registration database (D56 code); ≥8 blood transfusions (BT) within any 12-month period. Descriptive analyses included patients’ clinical characteristics, BT episodes, iron chelation therapy (ICT) use and healthcare resource utilization. RESULTS: A total of 448 patients with TDT were included in the analysis (49% male), of which 31% were diagnosed during the study period. Median age at first occurrence of diagnosis was 4 (0-47) years old. Of the 140 patients diagnosed after 2007, both first BT and first ICT use occurred at a median age of 5 (BT: 0-40; ICT: 1-42) years old. Across all patients, average number of BT per patient per year was 11±5, and 23% of patients had BT-related complications, in particular ‘Adverse event due to blood and its products in therapeutic use’ (68%). Almost all patients required ICT (96%), and combination therapy was used by 21% of them. Average number of hospitalizations was 13±6 per patient per year. BT accounted for 86% of the total hospitalization records. A total of 376 (84%) patients had ≥1 overnight hospitalization, with a median length of stay of 7 days. Almost all patients (99%) required ≥1 outpatient visit during the study period; 81% had ≥1 Emergency Room visit (with or without subsequent hospitalization). All-cause mortality rate was 7.1% (n=32) over study period, and death median age was 37 (5-55) years old. CONCLUSIONS: In France, TDT is associated with high clinical and economic burden.
Conference/Value in Health Info
2019-11, ISPOR Europe 2019, Copenhagen, Denmark
Code
PRO58
Topic
Economic Evaluation, Health Service Delivery & Process of Care
Topic Subcategory
Disease Management, Treatment Patterns and Guidelines
Disease
Rare and Orphan Diseases