title: Hydroxyurea and sickle cell anemia: effect on quality of life.
abstract: BACKGROUND
  The Multicenter Study of Hydroxyurea (HU) in Sickle Cell Anemia (MSH) previously showed that daily oral HU reduces painful sickle cell (SS) crises by 50% in patients with moderate to severe disease. The morbidity associated with this disease is known to have serious negative impact on the overall quality of life(QOL) of affected individuals.
  
  
  METHODS
  The data in this report were collected from the 299 patients enrolled in the MSH. Health quality of llife (HQOL) measures were assessed in the MSH as a secondary endpoint to determine if the clinical benefit of HU could translate into a measurable benefit perceptible to the patients. HQOL was assessed with the Profile of Mood States, the Health Status Short Form 36 (SF-36), including 4-week pain recall, and the Ladder of Life, self-administered twice 2-weeks apart pre-treatment and every 6 months during the two-year, randomized, double-blind, treatment phase. The effects of factors including randomized treatment, age, gender, pre-treatment crises frequency, Hb-F level mean, daily pain from 4-week pre-treatment diaries, and 2-year Hb-F response level (low or high) were investigated.
  
  
  RESULTS
  Over two years of treatment, the benefit of HU treatment on QOL, other than pain scales, was limited to those patients taking HU who maintained a high HbF response, compared to those with low HbF response or on placebo. These restricted benefits occurred in social function, pain recall and general health perception. Stratification according to average daily pain prior to treatment showed that responders to HU whose average daily pain score was 5-9 (substantial pain) achieved significant reduction in the tension scale compared to the placebo group and to non-responders. HU had no apparent effect on other QOL measures.
  
  
  CONCLUSIONS
  Treatment of SS with HU improves some aspects of QOL in adult patients who already suffer from moderate-to-severe SS.
authors.affiliation: Cardeza Foundation, Department of Medicine, Jefferson Medical College, Philadelphia, PA, USA. samir.ballas@jefferson.edu
authors.forename: Samir K
authors.initials: SK
authors.lastname: Ballas
authors.forename: Franca B
authors.initials: FB
authors.lastname: Barton
authors.forename: Myron A
authors.initials: MA
authors.lastname: Waclawiw
authors.forename: Paul
authors.initials: P
authors.lastname: Swerdlow
authors.forename: James R
authors.initials: JR
authors.lastname: Eckman
authors.forename: Charles H
authors.initials: CH
authors.lastname: Pegelow
authors.forename: Mabel
authors.initials: M
authors.lastname: Koshy
authors.forename: Bruce A
authors.initials: BA
authors.lastname: Barton
authors.forename: Duane R
authors.initials: DR
authors.lastname: Bonds
mesh_terms: D000203:Activities of Daily Living; D000328:Adult; D000755:Anemia, Sickle Cell / Q000503:physiopathology* / Q000523:psychology; D000986:Antisickling Agents / Q000627:therapeutic use*; D001772:Blood Cell Count; D004311:Double-Blind Method; D004909:Erythrocyte Indices / Q000187:drug effects; D005260:Female; D005319:Fetal Hemoglobin / Q000032:analysis / Q000187:drug effects; D006801:Humans; D006918:Hydroxyurea / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D010146:Pain / Q000188:drug therapy* / Q000209:etiology; D010147:Pain Measurement; D011788:Quality of Life / Q000523:psychology*; D018434:Sickness Impact Profile; D016896:Treatment Outcome
chemical_list: D000986:Antisickling Agents; D005319:Fetal Hemoglobin; D006918:Hydroxyurea