Hydroxyurea and sickle cell anemia: effect on quality of life.
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.
Ballas
Samir K
SK
Cardeza Foundation, Department of Medicine, Jefferson Medical College, Philadelphia, PA, USA. samir.ballas@jefferson.edu
Barton
Franca B
FB
Waclawiw
Myron A
MA
Swerdlow
Paul
P
Eckman
James R
JR
Pegelow
Charles H
CH
Koshy
Mabel
M
Barton
Bruce A
BA
Bonds
Duane R
DR
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
D000986:Antisickling Agents; D005319:Fetal Hemoglobin; D006918:Hydroxyurea