Abstract
Background
Myelofibrosis (MF) is a rare malignancy marked by splenomegaly, symptoms and anemia. Although Janus kinase inhibitors (JAKi) are central to MF therapy, many patients require additional anemia management, particularly red blood cell transfusions (RBC‑T). These impose substantial patient burden, yet their economic impact is sparsely investigated. This study estimates RBC‑T-related costs in anemic MF patients treated with JAKi in Germany from the payer perspective.
Methods
Nationwide, anonymized administrative claims data from 2014-2024 were retrospectively analysed. Eligible patients had an MF diagnosis (ICD‑10‑GM D47.1, D47.4, C94.6), received ruxolitinib or fedratinib, and ≥1 anemia treatment within 100 days before or during JAKi therapy. The index date was the first anemia therapy. Mean annual therapy costs (ATC) per patient included outpatient and inpatient costs for RBC-T and iron chelation therapy (ICT). Outpatient RBC-T units were identified via EBM codes and inpatient units via OPS codes. RBC-T unit counts were linked to estimated external unit, service and laboratory costs. ICT costs were directly obtained from claims. Subgroup analyses were conducted by JAKi therapy line and transfusion status.
Results
In total, 157 patients were included (mean age 72.7 years; 70.1% male). Overall RBC-T-related ATC (standard deviation) per patient were €5,936 (€2,402). ATC varied across subgroups: patients treated with ruxolitinib incurred €5,806 (€2,396), while those receiving fedratinib after prior ruxolitinib had €9,374 (€2,732). Transfusion‑dependent patients showed the highest ATC (€11,882 [€3,543]), followed by transfusion‑requiring (€3,904 [€1,196]) and transfusion‑independent patients (€910 [€371]).
Conclusions
The economic burden related to RBC-T in anemic MF patients treated with ruxolitinib or fedratinib is substantial. Results highlight the need for therapies that effectively address anemia in MF to help reduce associated resource utilization.
Key messages
• Transfusions impose a high economic burden in anemic myelofibrosis patients on JAK inhibitors, with greatest costs in second‑line fedratinib and transfusion‑dependent patients.
• The high transfusion burden underscores the need for therapies that better manage anemia and lessen reliance on transfusion-related resources.