Hydroxyurea and sickle cell anemia: effect on quality of life

Samir K Ballas, Franca B Barton, Myron A Waclawiw, Paul Swerdlow, James R Eckman, Charles H Pegelow, Mabel Koshy, Bruce A Barton, Duane R Bonds, Samir K Ballas, Franca B Barton, Myron A Waclawiw, Paul Swerdlow, James R Eckman, Charles H Pegelow, Mabel Koshy, Bruce A Barton, Duane R Bonds

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.

Conclusion: Treatment of SS with HU improves some aspects of QOL in adult patients who already suffer from moderate-to-severe SS.

Figures

Figure 1
Figure 1
Selected parameters from MOS Short-Form SF-36 Health Quality of Life and the Profile of Mood States in adults with moderate-severe sickle cell anemia, treated with hydroxyurea (solid line) or placebo (dashed line) for two years. Mean quality of life measures and the standard error of the mean are shown.
Figure 2
Figure 2
Selected parameters from MOS Short-Form SF-36 Health Quality of Life and the Profile of Mood States in adults with moderate-severe sickle cell anemia, treated with hydroxyurea or placebo for two years, and classified according to 2-year Hb-F response or placebo (short dashes). High responders (solid line) were above the 50th percentile of HbF change from baseline to 2-years in the hydroxyurea group; low responders (long dashes) were below the 50th percentile. Mean quality of life measures and the standard error of the mean are shown.
Figure 3
Figure 3
Distribution of number of annual crises in 299 MSH patients at trial entry.
Figure 4
Figure 4
Distribution of average daily pain from two-week diaries at trial entry.

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Source: PubMed

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