Subcutaneous immunoglobulin (SCIG) for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (CIDP), a multicenter randomized double-blind placebo-controlled trial: The PATH Study
2018; Elsevier BV; Volume: 130; Issue: 1 Linguagem: Inglês
10.1016/j.clinph.2018.09.067
ISSN1872-8952
AutoresDario Cocito, Erdita Peci, Giuseppe Lauria, Patrizia Dacci, Antonio Di Muzio, Roberta Telese, Angelo Schenone, Luana Benedetti, Giovanni Antonini, Stefania Morino, Sandro Sorbi, Sabrina Matà, Vera Bril, Nan van Geloven, Hans‐Peter Hartung, Richard A. Lewis, Gen Sobue, J.-P. Lawo, Orell Mielke, Billie L. Durn, David R. Cornblath, Ingemar S. J. Merkies, Ivo N. van Schaik,
Tópico(s)Myasthenia Gravis and Thymoma
ResumoSeveral CIDP patients need long-term corticosteroids or intravenous immunoglobulin (IVIG), with IVIG being associated with improved safety profile. SCIG is an alternative option for immunoglobulin delivery but it was not investigated in large-scale trials in CIDP. PATH was a randomized, double-blind trial investigating 0.2 and 0.4 g/kg weekly doses of SCIG IgPro20 (Hizentra®, CSL Behring) versus placebo for maintenance treatment in 172 CIDP patients. IVIG-dependent adults with definite or probable CIDP were eligible. The primary outcome was the percentage of subjects with a CIDP relapse (1-point deterioration on adjusted INCAT disability score) or who were withdrawn for any reason during the 24-week SCIg-treatment. Multiple secondary endpoints were assessed. Overall, 33% of patients on high-dose SCIG, 39% of those on low-dose SCIG and 63% of placebo recipients experienced CIDP relapse or were withdrawn from treatment (p < 0.05 for both SCIG doses vs placebo). INCAT score, MRC sum score, and grip strength remained stable with SCIG, while they deteriorated with placebo. High-dose SCIG prevented R-ODS decline. Adverse events occurred in 47 (27%) patients (18% placebo, 30% low-dose, and 35% high-dose). Both IgPro20 doses were effective and safe as maintenance treatment in patients with CIDP, compared with placebo.
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