{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,9]],"date-time":"2026-10-09T20:08:47Z","timestamp":1791576527590,"version":"4.3.4"},"reference-count":18,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,11,3]],"date-time":"2021-11-03T00:00:00Z","timestamp":1635897600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Br J Haematol"],"published-print":{"date-parts":[[2022,2]]},"abstract":"<jats:title>Summary<\/jats:title>\n                  <jats:p>\n                    We describe our single institution experience with cladribine therapy in 42 patients with systemic mastocytosis (SM): 22 advanced (adv\u2010SM; median age 65\u2009years, 68% males) and 20 indolent\/smouldering SM (ISM\/SSM; median age 56\u2009years, 45% males); subcategories included eight aggressive, 13 associated with another haematological neoplasm, one mast cell leukaemia, 17 ISM and three SSM. Overall\/major response rates were 77%\/45% for adv\u2010SM and 70%\/60% for ISM\/SSM, and median (range) duration of response 10\u2009(4\u201375) and 46\u2009(4\u2013140)\u2009months respectively. A &gt;50% reduction in bone marrow mast cell burden and serum tryptase level was documented in 63% and 67% of patients with adv\u2010SM and 50% and 46% with ISM\/SSM respectively. The presence of KIT proto\u2010oncogene, receptor tyrosine kinase (\n                    <jats:italic>KIT<\/jats:italic>\n                    )\n                    <jats:sup>D816V<\/jats:sup>\n                    predicted response in adv\u2010SM: 17 (90%) of 19 with and none of three without the mutation responded (\n                    <jats:italic>P<\/jats:italic>\n                    \u2009&lt;\u20090\u00b701). Treatment\u2010emergent adverse events were mostly limited to transient cytopenias: Grade 3\/4 neutropenia, thrombocytopenia, or lymphopenia occurred in 27%, 27% and 27% of patients with adv\u2010SM, and 5%, 5% and 30% with ISM\/SSM respectively. The present study provides practical information that might be considered when making treatment choices between cladribine and newer KIT\u2010targeted therapies and identifies the absence of\n                    <jats:italic>KIT<\/jats:italic>\n                    <jats:sup>D816V<\/jats:sup>\n                    as a potential marker of cladribine resistance in advanced SM; the latter observation needs confirmation in a larger study.\n                  <\/jats:p>","DOI":"10.1111\/bjh.17932","type":"journal-article","created":{"date-parts":[[2021,11,3]],"date-time":"2021-11-03T03:50:31Z","timestamp":1635911431000},"page":"975-983","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":24,"title":["Cladribine therapy for advanced and indolent systemic mastocytosis: Mayo Clinic experience in 42 consecutive cases"],"prefix":"10.1111","volume":"196","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4605-3821","authenticated-orcid":false,"given":"Ayalew","family":"Tefferi","sequence":"first","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaya","family":"Kittur","sequence":"additional","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Faiqa","family":"Farrukh","sequence":"additional","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kebede H.","family":"Begna","sequence":"additional","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mrinal M.","family":"Patnaik","sequence":"additional","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0824-3715","authenticated-orcid":false,"given":"Aref","family":"Al\u2010Kali","sequence":"additional","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle A.","family":"Elliott","sequence":"additional","affiliation":[{"name":"Division of Hematology Department of Internal Medicine Mayo Clinic Rochester MN USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaaren K.","family":"Reichard","sequence":"additional","affiliation":[{"name":"Division of Hematopathology Department of Laboratory Medicine Mayo Clinic Rochester MN 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