Fatma Jendoubi1, Nicolas Gaudenzio2, Adeline Gallini3, Mathilde Negretto1, Carle Paul1, Cristina Bulai Livideanu1. 1. Department of Dermatology, Mastocytosis National Reference Center (CEREMAST), Toulouse University Hospital, Toulouse, France. 2. UDEAR - Hôpital Purpan, UMR 1056 INSERM - University of Toulouse, Toulouse, France. 3. Department of Epidemiology and Public Health, UMR1027, INSERM-University of Toulouse, Toulouse, France.
Abstract
BACKGROUND: Mastocytosis is associated with mast cell (MC) mediator-related symptoms for which limited therapies are available. OBJECTIVE: Our aim was to assess the efficacy and safety of omalizumab in the treatment of MC mediator-related symptoms in adult patients with mastocytosis. RESULTS: We identified one multi-centre retrospective cohort study (39 patients), one retrospective cohort study (13 patients), 4 case series and 10 case reports. No published controlled randomized study was identified. We included 69 patients (13 patients with cutaneous mastocytosis and 56 with systemic mastocytosis). The mean age was 48 years. Omalizumab maintenance dose was 300 mg for the majority of patients. The mean duration of treatment was 17 months. Treatment led to a tolerability of venom immunotherapy and to a complete resolution of severe reactions in all patients with post-honeybee sting anaphylaxis. Complete resolution of idiopathic anaphylaxis episodes was noted in 84% of the patients. Complete resolution of palpitations, gastrointestinal, cutaneous, neuropsychiatric, respiratory and musculoskeletal symptoms was observed at a rate of 43%, 29%, 27%, 11%, 9% and 0%, respectively. Efficacy was maintained for the entire duration of the treatment in all but four responders. Adverse events were reported for 13 patients. CONCLUSIONS AND CLINICAL RELEVANCE: Omalizumab appears to prevent some life-threatening reactions associated with mastocytosis and may be a good option to treat the associated symptoms. However, the evidence relied upon is observational, uncontrolled and from a small number of patients. A randomized controlled trial is needed to better understand the place of omalizumab in mastocytosis treatment.
BACKGROUND:Mastocytosis is associated with mast cell (MC) mediator-related symptoms for which limited therapies are available. OBJECTIVE: Our aim was to assess the efficacy and safety of omalizumab in the treatment of MC mediator-related symptoms in adult patients with mastocytosis. RESULTS: We identified one multi-centre retrospective cohort study (39 patients), one retrospective cohort study (13 patients), 4 case series and 10 case reports. No published controlled randomized study was identified. We included 69 patients (13 patients with cutaneous mastocytosis and 56 with systemic mastocytosis). The mean age was 48 years. Omalizumab maintenance dose was 300 mg for the majority of patients. The mean duration of treatment was 17 months. Treatment led to a tolerability of venom immunotherapy and to a complete resolution of severe reactions in all patients with post-honeybee sting anaphylaxis. Complete resolution of idiopathic anaphylaxis episodes was noted in 84% of the patients. Complete resolution of palpitations, gastrointestinal, cutaneous, neuropsychiatric, respiratory and musculoskeletal symptoms was observed at a rate of 43%, 29%, 27%, 11%, 9% and 0%, respectively. Efficacy was maintained for the entire duration of the treatment in all but four responders. Adverse events were reported for 13 patients. CONCLUSIONS AND CLINICAL RELEVANCE: Omalizumab appears to prevent some life-threatening reactions associated with mastocytosis and may be a good option to treat the associated symptoms. However, the evidence relied upon is observational, uncontrolled and from a small number of patients. A randomized controlled trial is needed to better understand the place of omalizumab in mastocytosis treatment.
Authors: Jason Gotlib; Tracy I George; Melody C Carter; K Frank Austen; Bruce Bochner; Daniel F Dwyer; Jonathan J Lyons; Matthew J Hamilton; Joseph Butterfield; Patrizia Bonadonna; Catherine Weiler; Stephen J Galli; Lawrence B Schwartz; Hanneke Oude Elberink; Anne Maitland; Theoharis Theoharides; Celalettin Ustun; Hans-Peter Horny; Alberto Orfao; Michael Deininger; Deepti Radia; Mohamad Jawhar; Hanneke Kluin-Nelemans; Dean D Metcalfe; Michel Arock; Wolfgang R Sperr; Peter Valent; Mariana Castells; Cem Akin Journal: J Allergy Clin Immunol Date: 2021-03-11 Impact factor: 14.290
Authors: Peter Valent; Cem Akin; Karin Hartmann; Gunnar Nilsson; Andreas Reiter; Olivier Hermine; Karl Sotlar; Wolfgang R Sperr; Luis Escribano; Tracy I George; Hanneke C Kluin-Nelemans; Celalettin Ustun; Massimo Triggiani; Knut Brockow; Jason Gotlib; Alberto Orfao; Petri T Kovanen; Emir Hadzijusufovic; Irina Sadovnik; Hans-Peter Horny; Michel Arock; Lawrence B Schwartz; K Frank Austen; Dean D Metcalfe; Stephen J Galli Journal: Theranostics Date: 2020-08-29 Impact factor: 11.556
Authors: Magdalena Lange; Karin Hartmann; Melody C Carter; Frank Siebenhaar; Ivan Alvarez-Twose; Inés Torrado; Knut Brockow; Joanna Renke; Ninela Irga-Jaworska; Katarzyna Plata-Nazar; Hanna Ługowska-Umer; Justyna Czarny; Anna Belloni Fortina; Francesca Caroppo; Roman J Nowicki; Bogusław Nedoszytko; Marek Niedoszytko; Peter Valent Journal: Int J Mol Sci Date: 2021-03-04 Impact factor: 5.923