The Disability Index of the Health Assessment Questionnaire is a predictor and correlate of outcome in the high-dose versus low-dose penicillamine in systemic sclerosis trial

Abstract

Objective To explore the clinical implications of a score of ≥1.0 on the Disability Index of the Health Assessment Questionnaire (HAQ DI) at the first patient visit, and to examine the implications of improvement in HAQ DI score over 2 years in a cohort of systemic sclerosis (SSc) patients with diffuse cutaneous scleroderma. Methods SSc skin and visceral involvement was assessed in 134 SSc patients with diffuse scleroderma (mean ± SD disease duration of 10 ± 4 months) when they entered a multicenter drug trial and again 2 years later. Mortality and the occurrence of scleroderma renal crisis were assessed for a mean ± SD of 4.0 ± 1.1 years. Logistic and linear regression analyses were used to examine the relationship of the baseline HAQ DI score to morbidity, mortality, and visceral involvement, as well as the relationship of changes in the HAQ DI score to changes in physical examination, laboratory, and functional variables over 2 years. Results A baseline HAQ DI score of ≥1.0 was predictive of mortality (odds ratio 3.22, 95% confidence interval 1.097–9.468) over 4 years. Multivariate linear regression demonstrated that a model which included the erythrocyte sedimentation rate at baseline (P = 0.005) and changes at 2 years in the swollen joint count (P = 0.002), total skin score (P = 0.005), and white blood cell count (P = 0.005) best explained the change in HAQ DI score over 2 years (R2 = 0.528). The HAQ DI score and total skin score at baseline were highly correlated (correlation coefficient 0.368), as were changes in the HAQ DI score and the total skin score over 2 years (correlation coefficient 0.492). Although the HAQ DI score was heavily influenced by hand dysfunction at baseline and at 2 years, improvement (reduction) in the HAQ DI score over 2 years was related to factors other than hand dysfunction. Conclusion A baseline HAQ DI score of ≥1.0 predicted mortality over 4 years. Improvement in the HAQ DI score in these patients with diffuse scleroderma was associated with improvement in skin thickening, hand function, oral aperture, lung function, signs of arthritis, serum creatinine level, and the investigator's global assessment of improvement. The HAQ DI is a self‐administered questionnaire that SSc patients can complete easily and rapidly and that gives the practicing physician important information about prognosis, patient status, and changes in disease course over time.

Health values of patients with systemic sclerosis

“…The HAQ DI has been used extensively in persons with SSc (11,20,21). The overall HAQ DI score is determined by summing the highest item score in each of the 8 domains and dividing the sum by 8, yielding a score ranging from 0 (no disability) to 3 (severe disability).…”

“…The overall HAQ DI score is determined by summing the highest item score in each of the 8 domains and dividing the sum by 8, yielding a score ranging from 0 (no disability) to 3 (severe disability). In the original HAQ DI, an additional grade of difficulty was added for persons using assistive/adaptive devices (such as a cane or walker); however, as in more recent studies (21,22), we did not modify subjects' responses for use of assistive/adaptive devices.…”

High-dose immunosuppressive therapy and autologous hematopoietic cell transplantation for severe systemic sclerosis: long-term follow-up of the US multicenter pilot study

“…These evaluations included a medical history, physical examination, complete blood count, serum chemistries, autoantibodies (Scl-70, ANA), thyroid function tests, validated standard measurements of the mRSS, pulmonary function testing for FVC and DLCO adj , high-resolution computed tomography (HRCT) scans of the chest, multigated acquisition (MUGA) scan of the heart or echocardiogram for left ventricular ejection fraction (LVEF), and a modified Health Assessment Questionnaire Disability Index (mHAQ-DI). 5,[17][18][19][20][21] An ophthalmologic examination was done at the last study evaluation to assess for cataracts. In a subset of consenting patients, skin biopsies were done after HDIT next to the site of the baseline biopsy.…”

“…Days to neutrophil count above 500/L † 9 (7)(8)(9)(10)(11)(12)(13) Days to platelet count above 20 000/L ‡ 11 (7)(8)(9)(10)(11)(12)(13)(14)(15)(16)(17)(18)(19)(20)(21)(22)(23)(24)(25) *Minor complications observed infrequently during mobilization included edema, increased skin tightness, erythema, and arthralgias, but these resolved after G-CSF was stopped. All grafts were collected with a single course of G-CSF except for 1 patient who had discontinued mycophenolate mofetil immediately before starting collection of peripheral blood stem cells.…”

Predictors and outcomes of scleroderma renal crisis: Data from the high‐dose versus low‐dose D‐penicillamine in early diffuse systemic sclerosis trial

“…This RCT failed to document efficacy of an accepted therapy, yet few, if any, previous RCTs in SSc have included such a large number of patients. The authors should be congratulated for once again "mining" data from this "failed" trial to deduce pragmatic lessons about SSc (6)(7)(8)(9).…”