A prospective study of iron overload management in allogeneic hematopoietic cell transplantation survivors.
We report the results of a single-center, prospective evaluation for iron overload and subsequent treatment in 147 adult allogeneic hematopoietic cell transplantation (HCT) recipients who survived beyond 1 year after transplantation. Patients were screened by serum ferritin level; those with ferritin >1000 ng/mL underwent liver R2 magnetic resonance imaging to estimate liver iron concentration (LIC; normal < or =1.8 mg/g). Patients with significant iron overload (defined as LIC > or =5 mg/g), based on physician and patient preference, were offered observation only, phlebotomy, or enrollment in a pilot study of deferasirox. Sixteen patients had significant iron overload. Their median age was 51 years (range, 29-64 years), and they had survived a median of 21 months (range, 12-114 months). All 16 patients were transfusion-independent at study enrollment. Five patients received no treatment (median LIC, 6.4 mg/g; range, 5.1-28.3 mg/g), 8 underwent phlebotomy (median LIC, 13.1 mg/g; range, 7.8-43.0 mg/g), and 3 received daily deferasirox 20 mg/kg/day orally for 6 months (LIC, 6.3, 9.0, and 19.9 mg/g). Two patients had abnormal liver function tests, and 1 patient each had cirrhosis and unexplained congestive heart failure; all 4 of these patients underwent phlebotomy. Follow-up serum ferritin concentrations decreased spontaneously in 4 patients in the observation-only arm. Phlebotomy was generally well tolerated. Deferasirox also was well tolerated and led to decreased LIC after 6 months of therapy in all 3 patients. Phlebotomy is feasible in the majority of allogeneic HCT recipients who have survived for > or =1 year after HCT and have significant iron overload. Although the number of subjects is small, deferasirox may be a safe and effective alternative for allogeneic HCT survivors with iron overload who cannot undergo phlebotomy.
Majhail
Navneet S
NS
Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota 55455, USA. majha001@umn.edu
Lazarus
Hillard M
HM
Burns
Linda J
LJ
D000328:Adult; D001565:Benzoates / Q000009:adverse effects / Q000627:therapeutic use; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006321:Heart / Q000503:physiopathology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006454:Hemoglobins / Q000378:metabolism; D006801:Humans; D007501:Iron / Q000032:analysis; D019190:Iron Overload / Q000097:blood / Q000209:etiology / Q000503:physiopathology / Q000628:therapy*; D008099:Liver / Q000737:chemistry / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D018962:Phlebotomy / Q000009:adverse effects / Q000379:methods; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D014230:Triazoles / Q000009:adverse effects / Q000627:therapeutic use
D001565:Benzoates; D006454:Hemoglobins; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox