Conventional synthetic disease-modifying antirheumatic drugs and tumour necrosis factor inhibitors were ineffective or poorly tolerated, whereas tofacitinib and upadacitinib induced rapid and durable improvement across articular, cutaneous, vascular and pulmonary domains. These observations support a role for JAK-STAT signalling in MRH and suggest JAK inhibition as a rational therapeutic option in refractory multisystem disease.

P2, N=42, Recruiting, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

P2/3, N=192, Not yet recruiting, GlaxoSmithKline

P2, N=82, Recruiting, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

To report a case of AA developing during golimumab and leflunomide treatment for seropositive rheumatoid arthritis, with subsequent improvement following initiation of selective Janus kinase 1 (JAK1) inhibition...Upadacitinib was initiated for rheumatoid arthritis management and escalated from 15 mg to 30 mg...Although spontaneous remission and delayed corticosteroid effects cannot be excluded, the timing and magnitude of improvement support a temporal association with JAK1 inhibition. This case highlights a pragmatic therapeutic consideration when alopecia arises during TNF-α inhibitor therapy.

P1/2, N=20, Recruiting, University of Michigan Rogel Cancer Center | N=39 --> 20

P1, N=19, Completed, Imperial College London | Active, not recruiting --> Completed

P1, N=64, Not yet recruiting, Suzhou Zelgen Biopharmaceuticals Co.,Ltd

P1/2, N=24, Recruiting, University of Colorado, Denver | Not yet recruiting --> Recruiting | Initiation date: Jan 2026 --> Apr 2026

Ruxolitinib-based regimens, particularly combined with azacitidine, showed acceptable activity in AP (median OS 27.2 months). IC carried high rates of febrile neutropenia and sepsis; venetoclax was associated with prolonged cytopenias. This study confirms the poor prognosis of MPN-AP/BP, the absence of a unified UK consensus approach and the need for improved therapies and prospective studies to determine optimal treatment approaches for this challenging cohort.

P2, N=22, Active, not recruiting, University of Zurich | Recruiting --> Active, not recruiting

P2, N=120, Recruiting, Incyte Corporation | Active, not recruiting --> Recruiting