Get access

Treatment of transfusional iron overload in patients with myelodysplastic syndrome or severe anemia: data from multicenter clinical practices

Authors

  • Anastasios Raptis,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Mei Sheng Duh,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Si-Tien Wang,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Ellison Dial,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Ilias Fanourgiakis,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Barry Fortner,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Carole Paley,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Nikita Mody-Patel,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Mitra Corral,

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author
  • Jeffrey Scott

    1. From the University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania; Analysis Group, Inc., Boston, Massachusetts; Novartis Pharmaceuticals Corporation, Florham Park, New Jersey; and P4 Healthcare, Ellicott City, Maryland.
    Search for more papers by this author

  • This research was sponsored by Novartis Pharmaceuticals Corporation, East Hanover, NJ.

Mei Sheng Duh, MPH, ScD, Analysis Group, Inc., 111 Huntington Avenue, Tenth Floor, Boston, MA 02199; e-mail: mduh@analysisgroup.com.

Abstract

BACKGROUND: Patients with myelodysplastic syndrome (MDS) or severe anemia requiring repeated red blood cell (RBC) transfusions risk developing transfusional iron overload, which can reduce survival. Iron chelation therapy (ICT) has been shown to improve survival and quality of life in patients; however, ICT utilization in clinical practices is not well understood.

STUDY DESIGN AND METHODS: Medical records of patients diagnosed with MDS or severe anemia at least 6 months before data extraction, aged at least 21 years at diagnosis, and who received at least one RBC transfusion were reviewed. ICT eligibility was defined as at least 20 units of RBCs transfused or at least two serum ferritin levels exceeding 1000 µg/L. Study endpoint was ICT treatment rate among ICT-eligible patients with lower-risk MDS (International Prognostic Scoring System [low or intermediate-1]; World Health Organization [refractory anemia {RA}, refractory anemia with ringed sideroblasts {RARS}, refractory cytopenia with multilineage dysplasia {RCMD}, refractory cytopenia with multilineage dysplasia and ringed sideroblasts, or 5q]; French-American-British [RA/RARS]).

RESULTS: Among 78 ICT-eligible patients with lower-risk MDS, 32 (41%) received ICT. At ICT initiation, treated patients received on average 13.3 transfusions (27.6 units) and mean first post-ICT initiation serum ferritin was twice the MDS Foundation recommendation at 1949 µg/L. Median overall survival for all ICT-eligible patients was significantly longer for those ICT-treated patients than untreated patients (8.7 years vs. 4.7 years, log-rank p = 0.02; multivariate hazard ratio 0.372, p = 0.03).

CONCLUSION: This study finds only 41% of ICT-eligible patients with lower-risk MDS received ICT in clinical practice, and treatment was initiated later than recommended. Receipt of ICT was associated with significantly longer survival.

Ancillary