Refined Treatment Response Criteria for Indolent Systemic Mastocytosis Proposed by the ECNM-AIM Consortium.
Polina Pyatilova, Cem Akin, Iván Alvarez-Twose, Michel Arock, Patrizia Bonadonna, Knut Brockow, Joseph H Butterfield, Sigurd Broesby-Olsen, Melody C Carter, Mariana Castells, Tracy I George, Jason Gotlib, Georg Greiner, Theo Gülen, Karin Hartmann, Olivier Hermine, Hans-Peter Horny, Mohamed Jawhar, Magdalena Lange, Jonathan J Lyons, Marcus Maurer, Dean D Metcalfe, Boguslaw Nedoszytko, Marek Niedoszytko, Alberto Orfao, Andreas Reiter, Juliana Schwaab, Karl Sotlar, Wolfgang R Sperr, Massimo Triggiani, Peter Valent, Frank Siebenhaar
The journal of allergy and clinical immunology. In practice 2022 AugIndolent 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 <30% to treatment. To conclude, we recommend the use of PROMs as primary outcome parameters to define treatment response in patients with ISM in clinical trials and in everyday clinical practice. Copyright © 2022. Published by Elsevier Inc.
Citation
Polina Pyatilova, Cem Akin, Iván Alvarez-Twose, Michel Arock, Patrizia Bonadonna, Knut Brockow, Joseph H Butterfield, Sigurd Broesby-Olsen, Melody C Carter, Mariana Castells, Tracy I George, Jason Gotlib, Georg Greiner, Theo Gülen, Karin Hartmann, Olivier Hermine, Hans-Peter Horny, Mohamed Jawhar, Magdalena Lange, Jonathan J Lyons, Marcus Maurer, Dean D Metcalfe, Boguslaw Nedoszytko, Marek Niedoszytko, Alberto Orfao, Andreas Reiter, Juliana Schwaab, Karl Sotlar, Wolfgang R Sperr, Massimo Triggiani, Peter Valent, Frank Siebenhaar. Refined Treatment Response Criteria for Indolent Systemic Mastocytosis Proposed by the ECNM-AIM Consortium. The journal of allergy and clinical immunology. In practice. 2022 Aug;10(8):2015-2024
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PMID: 35724950
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