Table 1 |
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Primary and secondary outcomes in three randomised controlled trials and corresponding effect sizes |
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| Reference |
Outcomes |
Effect size |
Safety |
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| 1. Hoyles, et al. [3], 2006 |
Follow-up after 1 year Primary endpoints: |
3 withdrawals in the active group due to side effects (1 patient had intolerable nausea
and 1 patient had abnormal findings on liver function tests during treatment with
AZA) and no withdrawals in the placebo group (NNH = 11) |
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| FVC: no change P = 0.08 versus placebo |
ES = 0.35 (CI -0.24, 0.94) |
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| DLCO: no change P = 0.64 versus placebo |
ES = -0.01 (CI -0.59, 0.58) |
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| TLC: no change P = 0.61 |
ES = 0.06 (CI -0.53, 0.64) |
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| FEV1: no change P = 0.16 |
ES = 0.28 (CI -0.31, 0.86) |
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| Kco: no change |
ES = 0.3 (CI -0.29, 0.88) |
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| Secondary endpoint: |
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| HRCT: trends toward to improve P = 0.39 |
NNT = 5 |
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| 2. Tashkin, et al. [2], 2006 |
Skin score: decreased |
ES = -0.24 (CI 0.09, -0.56) |
20 patients withdrew in the active group and 13 patients in the placebo group, mostly
due to adverse events or serious adverse events within 12 months. (NNH = 11.2) |
| Diffuse |
ES = -0.06 (CI 0.27, -0.38) |
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| Limited |
ES = -0.04 (CI 0.28, -0.37) |
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| Primary outcome: |
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| FVC: increased (P < 0.03) favouring Cyc |
ES = 0.14 (CI -0.18, 0.47) |
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| Secondary outcomes: |
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| TLC: increased (P = 0.026) favouring Cyc |
ES = 0.19 (CI -0.13, 0.52) |
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| DLCO: no change |
ES = -0.06 (CI -0.38, 0.27) |
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| SF-36 score: |
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| Physical: no change versus placebo |
ES = 0.24 (CI -0.09, 0.56) |
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| Mental: no change versus placebo |
ES = 0.27 (CI -0.06, 0.6) |
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| HAQ-DI: decreased |
ES = -0.37 (CI -0.04, -0.69) |
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| Transitional dyspnea score: improvement |
Not possible to calculate |
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| P < 0.001 |
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| 3. Nadashkevich, et al. [11], 2006 |
MRSS: decreased after 12 (P < 0.001) and 18 months (P < 0.01) of therapy in |
ES = -1.31 (CI -0.74, -1.85) after 6 months |
No withdrawals Adverse events on Cyc included hair loss, nausea, dyspepsia, and leucopenia. |
| Cyc group versus baseline; P < 0.001 versus controlled patients |
ES = -5.94 (CI -4.7, -7.03) after 12 months |
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| ES = -9.21 (CI -7.39, -10.79) after 18 months |
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| Attack frequency of Raynaud: decreased after 12 (P < 0.001) and 18 months (P < 0.01) of therapy in Cyc group versus baseline; P < 0.001 versus controlled patients |
ES = -1.99 (CI -1.34, -2.58) after 6 months |
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| ES = -8.35 (CI -6.69, -9.81) after 12 months |
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| ES = -11.68 (CI -9.41, -13.66) after 18 months NNT = 1.3 |
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| FVC: no change in Cyc group (P = NS), worsening in AZA group (P < 0.01) after 6, 12, and 18 months of therapy versus baseline; P < 0.001 versus controlled patients |
ES = 3.38 (CI 2.56, 4.12) after 6 months |
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| ES = 4.92 (CI 3.85, 5.86) after 12 months |
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| ES = 7.38 (CI 5.89, 8.68) after 18 months |
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| DLCO: no change in Cyc group (P = NS), worsening in AZA group (P < 0.01) after 6, 12, and 18 months of change in Cyc group (P = NS), worsening in AZA group (P < 0.001) after 12 and 18 months of therapy versus baseline; P < 0.001 versus controlled patients |
ES = 2.46 (CI 1.76, 3.1) after 6 months |
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| ES = 5.12 (CI 4.02, 6.09) after 12 months |
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| ES = 7.72 (CI 6.16, 9.07) after 18 months |
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AZA, azathioprine; CI, confidence interval; Cyc, cyclophosphamide; DLCO, diffusing lung capacity for carbon monoxide; ES, effect size; FEV1, forced expiratory volume in 1 second; FVC, forced vital capacity; HAQ-DI, Health Assessment Questionnaire-Disability Index; HRCT, high-resolution computed tomography; Kco, monoxide transfer coefficient; MRSS, modified Rodnan skin score; NNH, number needed to harm; NNT, number needed to treat; NS, not significant; SF-36, short form-36; TLC, total lung capacity. |
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Miniati et al. Arthritis Research & Therapy 2009 11:103 doi:10.1186/ar2576 |
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