医学
髓样
免疫学
鲁索利替尼
骨髓增生异常综合症
慢性粒单核细胞白血病
依那西普
内科学
细胞减少
肿瘤科
肿瘤坏死因子α
骨髓
骨髓纤维化
作者
Rahul Mishra,Cassandra Calabrese,Akriti Jain,Abhay Singh
标识
DOI:10.1016/j.leukres.2024.107584
摘要
Approximately one-third of patients with myeloid disorders like myelodysplastic syndrome (MDS) and chronic myelomonocytic leukemia (CMML) exhibit inflammatory and autoimmune disorders (IADs). These IADs often include atypical and incomplete forms of common autoimmune conditions, and exhibit resistance to conventional immunosuppressive therapies. There is growing interest in molecular relationships between IADs and MDS/CMML to find potential targeted therapies. Recently, patients with somatic mutations in the UBA1 gene were identified as having VEXAS syndrome. Herein, we present a concise case-series illustrating concurrent elderly-onset inflammatory manifestations and myeloid disorders (MDS, CMML, and idiopathic cytopenia of undetermined significance). These patients manifested inflammatory or autoimmune symptoms, including erythema nodosum, Raynaud's phenomenon, Sjogren syndrome, and refractory pruritus, having onset after 60-years of age. The inflammatory manifestations were largely refractory to traditional immunosuppressive regimens. Remarkably, treatment with a JAK-1 inhibitor, upadacitinib, in two cases yielded marked resolution of inflammatory symptoms, facilitating the gradual tapering of corticosteroids, improvement of hemoglobin levels, and reduction in serum C-reactive protein levels. Upon loss of response to upadacitinib, JAK-2 inhibitor ruxolitinib provided clinical benefit in one of the cases, facilitating further tapering of glucocorticoids. This arena warrants further exploration through prospective studies of larger cohorts to delineate optimal management strategies. • Myeloid disorders and IADs can often coexist. • These IADs are frequently resistant to standard immunosuppressive treatments. • JAK inhibitors may enable select patients to overcome this resistance. • Hemoglobin levels improved, and serum C-reactive protein levels decreased with the use of a JAK-inhibitor. • The use of a JAK inhibitor can serve as an effective steroid-sparing approach.
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