PURPOSE: Several immunoglobulin (IG) preparations have been approved for the immunomodulatory treatment of the neurological autoimmune diseases (AID) Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), and multifocal motor neuropathy (MMN). Although efficacy has been proven in randomised clinical trials, long-term outcome data on drug utilization, effectiveness, tolerability, health related quality of life, and economic variables are lacking. METHODS: In the prospective, observational internet-based SIGNS registry, patients of all age groups are eligible if they have received or are scheduled for IG therapy for neurological AID or primary or severe secondary immunodeficiency. RESULTS: Of the 306 patients currently included in the database (1 November 2011), 51 have neurological AID (27 males; mean age 56 ± 15 years): 21 CIDP, 7 MMN, 11 multiple sclerosis (MS), 6 myasthenia gravis, 2 myositis, 4 others (no cases of GBS). Mean duration of disease since first symptoms was 7.8 years, and disease duration since diagnosis was 5.9 years. Eight different IG preparations have been reported as current therapy. According to SF-36, patients' quality of life is substantially impaired. CONCLUSIONS: Present data indicate some off-label use of IG (e.g. in MS) in patients with neurological AID. Quality of life in these patients is substantially compromised. Increasing patient numbers and extended follow-up periods will provide data on treatment concepts and disease development in AID patients.
PURPOSE: Several immunoglobulin (IG) preparations have been approved for the immunomodulatory treatment of the neurological autoimmune diseases (AID) Guillain-Barré syndrome (GBS), chronic inflammatory demyelinating polyneuropathy (CIDP), and multifocal motor neuropathy (MMN). Although efficacy has been proven in randomised clinical trials, long-term outcome data on drug utilization, effectiveness, tolerability, health related quality of life, and economic variables are lacking. METHODS: In the prospective, observational internet-based SIGNS registry, patients of all age groups are eligible if they have received or are scheduled for IG therapy for neurological AID or primary or severe secondary immunodeficiency. RESULTS: Of the 306 patients currently included in the database (1 November 2011), 51 have neurological AID (27 males; mean age 56 ± 15 years): 21 CIDP, 7 MMN, 11 multiple sclerosis (MS), 6 myasthenia gravis, 2 myositis, 4 others (no cases of GBS). Mean duration of disease since first symptoms was 7.8 years, and disease duration since diagnosis was 5.9 years. Eight different IG preparations have been reported as current therapy. According to SF-36, patients' quality of life is substantially impaired. CONCLUSIONS: Present data indicate some off-label use of IG (e.g. in MS) in patients with neurological AID. Quality of life in these patients is substantially compromised. Increasing patient numbers and extended follow-up periods will provide data on treatment concepts and disease development in AID patients.
Authors: Wilhelm Kirch; Ralf Gold; Manfred Hensel; Maria Fasshauer; David Pittrow; Dörte Huscher; Marcel Reiser; Martin Stangel; Ulrich Baumann; Michael Borte Journal: Med Klin (Munich) Date: 2010-09-28
Authors: Elisabeth A Cats; W-Ludo van der Pol; Sanne Piepers; Nicoll C Notermans; R M van den Berg-Vos; L H van den Berg Journal: J Neurol Date: 2008-09-03 Impact factor: 4.849
Authors: I Elovaara; S Apostolski; P van Doorn; N E Gilhus; A Hietaharju; J Honkaniemi; I N van Schaik; N Scolding; P Soelberg Sørensen; B Udd Journal: Eur J Neurol Date: 2008-09 Impact factor: 6.089
Authors: Richard A C Hughes; Peter Donofrio; Vera Bril; Marinos C Dalakas; Chunqin Deng; Kim Hanna; Hans-Peter Hartung; Norman Latov; Ingemar S J Merkies; Pieter A van Doorn Journal: Lancet Neurol Date: 2008-02 Impact factor: 44.182
Authors: Cristian Vilos; Luis Constandil; Paula I Rodas; Mario Cantin; Katherine Zepeda; Natalia Herrera; Luis A Velasquez Journal: Drug Des Devel Ther Date: 2014-05-29 Impact factor: 4.162