Impact of oral cyclophosphamide on health-related quality of life in patients with active scleroderma lung disease: results from the scleroderma lung study.
OBJECTIVE
To assess the impact of cyclophosphamide (CYC) on the health-related quality of life (HRQOL) of patients with scleroderma after 12 months of treatment.


METHODS
One hundred fifty-eight subjects participated in the Scleroderma Lung Study, with 79 each randomized to CYC and placebo arms. The study evaluated the results of 3 measures of health status: the Short Form 36 (SF-36), the Health Assessment Questionnaire (HAQ) disability index (DI), and Mahler's dyspnea index, and the results of 1 preference-based measure, the SF-6D. The differences in the HRQOL between the 2 groups at 12 months were calculated using a linear mixed model. Responsiveness was evaluated using the effect size. The proportion of subjects in each treatment group whose scores improved at least as much as or more than the minimum clinically important difference (MCID) in HRQOL measures was assessed.


RESULTS
After adjustment for baseline scores, differences in the HAQ DI, SF-36 role physical, general health, vitality, role emotional, mental health scales, and SF-36 mental component summary (MCS) score were statistically significant for CYC versus placebo (P < 0.05). Effect sizes were negligible (<0.20) for all of the scales of the SF-36, HAQ DI, and SF-6D at 12 months. In contrast, a higher proportion of patients who received CYC achieved the MCID compared with placebo in the HAQ DI score (30.9% versus 14.8%), transitional dyspnea index score (46.4% versus 12.7%), SF-36 MCS score (33.3% versus 18.5%), and SF-6D score (21.3% versus 3.8%).


CONCLUSIONS
One year of treatment with CYC leads to an improvement in HRQOL in patients with scleroderma lung disease.
Khanna
Dinesh
D
University of Cincinnati, Cincinnati, Ohio 45267-0563, USA. Dinesh.Khanna@uc.edu
Yan
Xiaohong
X
Tashkin
Donald P
DP
Furst
Daniel E
DE
Elashoff
Robert
R
Roth
Michael D
MD
Silver
Richard
R
Strange
Charlie
C
Bolster
Marcy
M
Seibold
James R
JR
Riley
David J
DJ
Hsu
Vivien M
VM
Varga
John
J
Schraufnagel
Dean E
DE
Theodore
Arthur
A
Simms
Robert
R
Wise
Robert
R
Wigley
Fredrick
F
White
Barbara
B
Steen
Virginia
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Read
Charles
C
Mayes
Maureen
M
Parsley
Ed
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Mubarak
Kamal
K
Connolly
M Kari
MK
Golden
Jeffrey
J
Olman
Mitchell
M
Fessler
Barri
B
Rothfield
Naomi
N
Metersky
Mark
M
Clements
Philip J
PJ
D000328:Adult; D018501:Antirheumatic Agents / Q000627:therapeutic use*; D003520:Cyclophosphamide / Q000627:therapeutic use*; D004185:Disability Evaluation; D004311:Double-Blind Method; D005260:Female; D006305:Health Status Indicators; D006801:Humans; D008137:Longitudinal Studies; D008171:Lung Diseases / Q000188:drug therapy* / Q000503:physiopathology / Q000523:psychology; D008297:Male; D008875:Middle Aged; D011788:Quality of Life; D015203:Reproducibility of Results; D012595:Scleroderma, Systemic / Q000188:drug therapy* / Q000503:physiopathology / Q000523:psychology; D012720:Severity of Illness Index; D016896:Treatment Outcome
D018501:Antirheumatic Agents; D003520:Cyclophosphamide