Author(s)
Anastasios Raptis, MD, Co-Director, Myelodysplastic Syndrome Program1, Mei Sheng Duh, MPH, ScD, Vice President2, Si-Tien Wang, MS, Associate2, Ellison Dial, MBA, Associate2, Ilias Fanourgiakis, PhD, Medical Intern1, Barry Fortner, PhD, Sr. V.P., Scientific Affairs and Provider Services3, Carole Paley, MD, Sr. Medical Director4, Nikita Mody-Patel, PharmD, Assistant Director, HE&OR, NS4, Mitra Corral, MS, Assistant Director Health Economics & Outcomes Research, Onc US4, Jeffrey Scott, MD, President and Medical Director51University of Pittsburgh School of Medicine, Pittsburgh, PA, USA; 2 Analysis Group, Inc., Boston, MA, USA; 3 P4 Healthcare, Lakeland, TN, USA; 4 Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA; 5 P4 Healthcare, Ellicott City, MD, USA
OBJECTIVES Patients with myelodysplastic syndrome (MDS) or severe anemia requiring repeated transfusions of red blood cells (RBCs) risk developing transfusional iron overload (TIO), which can cause organ damage and reduce survival. Iron chelation therapy (ICT) has been shown to improve survival and quality of life in patients with TIO; however, ICT utilization in clinical practices is not well understood. METHODS The medical records of patients diagnosed with MDS or severe anemia ≥6 months before data extraction, aged ≥21 years at their diagnosis, received ≥1 RBC transfusion were reviewed. ICT-eligibility was defined as ≥20 units of RBCs transfused or ≥2 serum ferritin (SF) tests ≥1000 mcg/L. Study endpoint was ICT-treatment rate among ICT-eligible patients with lower-risk MDS [IPSS (low or intermediate-1); WHO (RA, RARS, RCMD, RCMD-RS or 5q); FAB (RA or RARS)]. Characteristics and survival of treated and untreated groups were described. RESULTS Medical records data for 283 patients were extracted. Among 78 ICT-eligible patients with lower-risk MDS, only 32 (41%) received ICT. At ICT-initiation, treated patients received on average 13.3 transfusions (27.6 units) and mean first SF near-ICT-initiation was twice the recommended level at 1949 mcg/L. Median overall survival for all ICT-eligible patients was significantly longer for those ICT-treated than untreated (8.7 versus 4.7 years, log-rank p=0.02; multivariate hazards ratio=0.372, p=0.03). CONCLUSIONS This observational study finds only 41% of ICT-eligible patients with lower-risk MDS received ICT in clinical practice, and their treatment was initiated later than recommended. Among all ICT-eligible patients, those who received ICT had a significantly better overall survival than untreated patients.
Conference/Value in Health Info
2009-05, ISPOR 2009, Orlando, FL, USA
Value in Health, Vol. 12, No. 3 (May 2009)
Code
PSY12
Topic
Clinical Outcomes
Topic Subcategory
Relating Intermediate to Long-term Outcomes
Disease
Systemic Disorders/Conditions