TY - JOUR
T1 - The Therapeutic Efficacy of Botulinum Toxin in Treating Scleroderma-Associated Raynaud's Phenomenon
T2 - A Randomized, Double-Blind, Placebo-Controlled Clinical Trial
AU - Bello, Ricardo J.
AU - Cooney, Carisa M.
AU - Melamed, Eitan
AU - Follmar, Keith
AU - Yenokyan, Gayane
AU - Leatherman, Gwendolyn
AU - Shah, Ami A.
AU - Wigley, Fredrick M.
AU - Hummers, Laura K.
AU - Lifchez, Scott D.
N1 - Publisher Copyright:
© 2017, American College of Rheumatology
PY - 2017/8
Y1 - 2017/8
N2 - Objective: To assess the therapeutic efficacy of local injections of botulinum toxin type A (Btx-A) in improving blood flow to the hands of patients with Raynaud's phenomenon (RP) secondary to scleroderma. Methods: In this randomized, double-blind, placebo-controlled clinical trial, patients with scleroderma-associated RP received Btx-A (50 units in 2.5 ml sterile saline) in one randomly selected hand and sterile saline (2.5 ml) in the opposite hand. Follow-up at 1 and 4 months postinjection included laser Doppler imaging of hands, patient-reported outcomes, and physical examination. We compared outcomes using paired t-tests and population-average generalized models with generalized estimating equations. Results: Of 40 patients enrolled, 25 had limited scleroderma and 15 had diffuse scleroderma. From baseline to 1-month follow-up, there was a greater reduction in average blood flow in Btx-A–treated hands compared to placebo-treated hands. The model estimated that this difference was statistically significant (average difference −30.08 flux units [95% confidence interval −56.19, −3.98], P for interaction = 0.024). This difference was mainly influenced by patients with longstanding RP and diffuse scleroderma. Change in blood flow at 4-month follow-up was not significantly different between groups. Clinical measures (QuickDASH, McCabe Cold Sensitivity Score, pain on a visual analog scale, and Raynaud's Condition Score) improved slightly for Btx-A–treated hands. Conclusion: Our laboratory-based laser Doppler imaging flow data do not support using Btx-A to treat RP in all scleroderma patients. The secondary clinical outcomes suggest some positive effect, but its clinical meaningfulness is questionable. The role of Btx-A in treating RP should be further studied with more homogeneous patient populations and in unique clinical situations such as acute digital ischemia.
AB - Objective: To assess the therapeutic efficacy of local injections of botulinum toxin type A (Btx-A) in improving blood flow to the hands of patients with Raynaud's phenomenon (RP) secondary to scleroderma. Methods: In this randomized, double-blind, placebo-controlled clinical trial, patients with scleroderma-associated RP received Btx-A (50 units in 2.5 ml sterile saline) in one randomly selected hand and sterile saline (2.5 ml) in the opposite hand. Follow-up at 1 and 4 months postinjection included laser Doppler imaging of hands, patient-reported outcomes, and physical examination. We compared outcomes using paired t-tests and population-average generalized models with generalized estimating equations. Results: Of 40 patients enrolled, 25 had limited scleroderma and 15 had diffuse scleroderma. From baseline to 1-month follow-up, there was a greater reduction in average blood flow in Btx-A–treated hands compared to placebo-treated hands. The model estimated that this difference was statistically significant (average difference −30.08 flux units [95% confidence interval −56.19, −3.98], P for interaction = 0.024). This difference was mainly influenced by patients with longstanding RP and diffuse scleroderma. Change in blood flow at 4-month follow-up was not significantly different between groups. Clinical measures (QuickDASH, McCabe Cold Sensitivity Score, pain on a visual analog scale, and Raynaud's Condition Score) improved slightly for Btx-A–treated hands. Conclusion: Our laboratory-based laser Doppler imaging flow data do not support using Btx-A to treat RP in all scleroderma patients. The secondary clinical outcomes suggest some positive effect, but its clinical meaningfulness is questionable. The role of Btx-A in treating RP should be further studied with more homogeneous patient populations and in unique clinical situations such as acute digital ischemia.
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U2 - 10.1002/art.40123
DO - 10.1002/art.40123
M3 - Article
C2 - 28426903
AN - SCOPUS:85021338096
SN - 2326-5191
VL - 69
SP - 1661
EP - 1669
JO - Arthritis and Rheumatology
JF - Arthritis and Rheumatology
IS - 8
ER -