Richard Lemal1, Guillemette Fouquet2, Louis Terriou3, Mélanie Vaes4, Cristina Bulai Livideanu5, Laurent Frenzel6, Stéphane Barete7, Danielle Canioni8, Ludovic Lhermitte9, Julien Rossignol10, Michel Arock11, Patrice Dubreuil12, Olivier Lortholary13, Olivier Hermine14. 1. Histocompatibility Laboratory, EA 7453-Université Clermont Auvergne, CHU de Clermont-Ferrand, Clermont-Ferrand, France. Electronic address: rlemal@chu-clermontferrand.fr. 2. Imagine Institute, INSERM U1163 and CNRS ERL 8654, Paris Descartes University, Sorbonne Paris Cité, Paris, France. 3. Department of Internal Medicine and Clinical Immunology, Hôpital Huriez, CHRU Lille, Lille, France. 4. Department of Hematology, Université Libre de Bruxelles, Hôpital Erasme, Brussels, Belgium; Department of Hematology, Université Libre de Bruxelles, CHU Tivoli, La Louvière, Belgium. 5. French Reference Center for Mastocytosis (CEREMAST), Department of Dermatology, Hôpital Larrey, CHU Toulouse, Toulouse, France. 6. Department of Hematology, Necker Children's Hospital, APHP, Paris, France. 7. French Reference Center for Mastocytosis (CEREMAST), Dermatology Department, Pitié-Salpêtrière Hospital, AP-HP, Paris, France. 8. Pathology Department, Necker Children's Hospital, APHP, Paris, France. 9. Hematology Department, Necker Children's Hospital, APHP, Paris, France. 10. French Reference Center for Mastocytosis (CEREMAST), Necker Children's Hospital, APHP, Paris, France; Hematology Department, Gustave Roussy, Université Paris-Saclay, Villejuif, France. 11. LBPA CNRS UMR8113, Ecole Normale Supérieure de Paris-Saclay, Cachan, France; Hematology Department, Pitié-Salpêtrière Hospital, APHP, Paris, France. 12. Centre de Recherche en Cancérologie de Marseille, Inserm U1068, Marseille, France. 13. Necker Pasteur Center for Infectious Diseases and Tropical Medicine, Necker Children's Hospital, APHP, Paris, France. 14. Imagine Institute, INSERM U1163 and CNRS ERL 8654, Paris Descartes University, Sorbonne Paris Cité, Paris, France; Department of Hematology, Necker Children's Hospital, APHP, Paris, France; French Reference Center for Mastocytosis (CEREMAST), Necker Children's Hospital, APHP, Paris, France.
Abstract
BACKGROUND: Patients with mast cell diseases may suffer from various distressing symptoms, which can be insufficiently controlled with available therapies, severely affecting their quality of life. There is a need for new and safe treatment options for these patients. OBJECTIVES: We aimed to evaluate safety and efficacy of omalizumab administration in patients with a symptomatic mast cell disorder. METHODS: We included 55 patients with a mast cell disorder associated with debilitating symptoms who received omalizumab treatment between January 2015 and December 2017, after a multidisciplinary team meeting at the French National Reference Center for Mastocytosis. RESULTS: A complete response was achieved for 1 patient (1.8%), a major response for 30 patients (54.5%), and a partial response for 12 patients (21.8%), resulting in an overall best response rate of 78.2% (43 of 55 patients). The response was persistent at least 3 months in 33 of 43 responding patients (76.7%). At the last follow-up, the final overall response rate was 58.2% (32 of 55 patients). Median time to first response was 2 months and median time to best response was 6 months. Omalizumab was dramatically effective on all superficial and general vasomotor symptoms and on most gastrointestinal or urinary symptoms, and partially effective on most neuropsychiatric symptoms. Safety profile was acceptable, except for one severe adverse event (edema of the larynx and dyspnea after the first injection of omalizumab). Side effects were reported in 16 patients (29%), mainly of low to mild intensity, yet causing interruption of treatment in 5 patients (9%). CONCLUSION: Omalizumab seems to be a useful therapeutic option to control mast cell-mediator symptoms and displays a favorable safety profile.
BACKGROUND:Patients with mast cell diseases may suffer from various distressing symptoms, which can be insufficiently controlled with available therapies, severely affecting their quality of life. There is a need for new and safe treatment options for these patients. OBJECTIVES: We aimed to evaluate safety and efficacy of omalizumab administration in patients with a symptomatic mast cell disorder. METHODS: We included 55 patients with a mast cell disorder associated with debilitating symptoms who received omalizumab treatment between January 2015 and December 2017, after a multidisciplinary team meeting at the French National Reference Center for Mastocytosis. RESULTS: A complete response was achieved for 1 patient (1.8%), a major response for 30 patients (54.5%), and a partial response for 12 patients (21.8%), resulting in an overall best response rate of 78.2% (43 of 55 patients). The response was persistent at least 3 months in 33 of 43 responding patients (76.7%). At the last follow-up, the final overall response rate was 58.2% (32 of 55 patients). Median time to first response was 2 months and median time to best response was 6 months. Omalizumab was dramatically effective on all superficial and general vasomotor symptoms and on most gastrointestinal or urinary symptoms, and partially effective on most neuropsychiatric symptoms. Safety profile was acceptable, except for one severe adverse event (edema of the larynx and dyspnea after the first injection of omalizumab). Side effects were reported in 16 patients (29%), mainly of low to mild intensity, yet causing interruption of treatment in 5 patients (9%). CONCLUSION:Omalizumab seems to be a useful therapeutic option to control mast cell-mediator symptoms and displays a favorable safety profile.
Authors: Michel Arock; Karl Sotlar; Jason Gotlib; Wolfgang R Sperr; Karin Hartmann; Lawrence B Schwartz; Cem Akin; Hans-Peter Horny; Peter Valent Journal: Leuk Lymphoma Date: 2019-12-26
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; Boguslaw Nedoszytko; Patrizia Bonadonna; Karin Hartmann; Marek Niedoszytko; Knut Brockow; Frank Siebenhaar; Massimo Triggiani; Michel Arock; Jan Romantowski; Aleksandra Górska; Lawrence B Schwartz; Dean D Metcalfe Journal: Int J Mol Sci Date: 2020-11-27 Impact factor: 5.923
Authors: G J Molderings; F L Dumoulin; J Homann; B Sido; J Textor; M Mücke; G J Qagish; R Barion; M Raithel; D Klingmüller; V S Schäfer; H J Hertfelder; D Berdel; G Tridente; L B Weinstock; L B Afrin Journal: Naunyn Schmiedebergs Arch Pharmacol Date: 2020-05-06 Impact factor: 3.000
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