1. Refined Treatment Response Criteria for Indolent Systemic Mastocytosis Proposed by the ECNM-AIM Consortium
- Author
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Pyatilova, Polina, Akin, Cem, Álvarez-Twose, Iván, Arock, Michel, Bonadonna, Patrizia, Brockow, Knut, Butterfield, Joseph H., Broesby-Olsen, Sigurd, Carter, Melody C., Castells, Mariana, George, Tracy I., Gotlib, Jason, Greiner, Georg, Gulen, Theo, Hartmann, Karin, Hermine, Olivier, Horny, Hans-Peter, Jawhar, Mohamad, Lange, Magdalena, Lyons, Jonathan J., Maurer, Marcus, Metcalfe, Dean D., Nedoszytko, Boguslaw, Niedoszytko, Marek, Orfao, Alberto, Reiter, Andreas, Schwaab, Juliana, Sotlar, Karl, Sperr, Wolfgang R., Triggiani, Massimo, Valent, Peter, Siebenhaar, Frank, National Institute of Allergy and Infectious Diseases (US), National Institutes of Health (US), Medical University of Gdańsk, Austrian Science Fund, and Publica
- Subjects
Mastocytosis, Systemic ,Mast cell activation ,Mast cells ,Mastocytosis ,Response criteria ,Signs ,Symptoms ,Quality of Life ,Humans ,Immunology and Allergy ,Tryptases - Abstract
Indolent systemic mastocytosis (ISM) has a favorable prognosis and normal life expectancy. However, many patients suffer from mast cell (MC) mediator-related symptoms, which significantly affect quality of life (QoL). Cutaneous, gastrointestinal, and neurological complaints, musculoskeletal pain, and the presence of skin lesions, anaphylaxis, and osteoporosis are the main symptoms and signs in ISM and must be assessed in all patients before and during treatment. Validated mastocytosis-specific patient-reported outcome measures (PROMs) should be used for this purpose. Serum tryptase and KIT D816V allele burden are recommended as secondary outcome parameters, noting that they do not reflect the severity of signs, symptoms, and related QoL impairment, but indirectly express MC burden. Changes from baseline of 90%, 60%, and 30% indicate complete response >90%, major response 60% to 90%, partial response 30% to 60%, and no response, M. C. Carter, J. J. Lyons, and D. D. Metcalfe were supported by the Division of Intramural Research, National Institutes of Allergic and Infectious Diseases, and National Institutes of Health. M. Niedoszytko was supported by the Medical University of Gdansk grant 02-0141/07/231. P. Valent was supported by the Austrian Science Fund (FWF) grant # P32470-B.
- Published
- 2022