Background Autologous haematopoietic stem cell transplantation (HSCT) improves survival in systemic sclerosis (SSc) with poor prognosis, but is hampered by treatment-related mortality (TRM).... Show moreBackground Autologous haematopoietic stem cell transplantation (HSCT) improves survival in systemic sclerosis (SSc) with poor prognosis, but is hampered by treatment-related mortality (TRM). Objective To evaluate event-free survival (EFS), TRM, response to treatment, disease progression and patient characteristics associated with events.Methods All patients treated with HSCT for SSc in The Netherlands until 2017 (n=92) were included. Data on skin involvement (modified Rodnan skin score (mRSS), pulmonary function (forced vital capacity (FVC) and diffusion capacity of the lungs for carbon monoxide (DLCO)), extent of interstitial lung disease on high-resolution CT using Goh scores and left ventricular ejection fraction (LVEF) were collected at baseline, 1, 2 and 5 years. Occurrence of events, defined as death or major organ failure, were collected until 2019. As control, a comparison between patients treated with cyclophosphamide (CYC) and patients with HSCT who participated in the Autologous Stem Cell Transplantation International Scleroderma (ASTIS) trial was performed.Results Median follow-up was 4.6 years. EFS estimates at 5, 10 and 15 years were 78%, 76% and 66%, respectively. Twenty deaths occurred. Mean FVC, DLCO, mRSS and Goh scores all improved significantly. Disease progression occurred in 22 patients. Frequency of TRM decreased over time and occurred more often in males. Events were independently associated with male sex, LVEF <50% and older age. In ASTIS, patients treated with HSCT (n=23) 7 events occurred versus 13 in the CYC group (n=22).Conclusion Our data confirm long-term efficacy of HSCT in improving survival, skin and lung involvement in SSc. Male sex, lower LVEF and older age at baseline were identified as risk factors for events. Show less
Mahler, E.A.M.; Cuperus, N.; Bijlsma, J.J.W.; Vlieland, T.P.M.V.; Hoogen, F.H.J. van den; Broeder, A.A. den; Ende, C.H. van den 2017
PURPOSE OF REVIEW: To summarize recent literature on nonpharmacological and nonsurgical interventions in patients with rheumatoid arthritis (RA). RECENT FINDINGS: Recent systematic reviews and... Show morePURPOSE OF REVIEW: To summarize recent literature on nonpharmacological and nonsurgical interventions in patients with rheumatoid arthritis (RA). RECENT FINDINGS: Recent systematic reviews and individual studies substantiate the effectiveness of aerobic and strength exercise programmes in RA. The evidence for the promotion of physical activity according to public health recommendations is scarce, and implementation research found that the reach and maintenance of exercise or physical activity programmes in RA patients are suboptimal. For self-management interventions, characteristics that increase their effectiveness were identified, including the use of cognitive behavioural approaches and approaches derived from the self-regulation theory. A limited number of recent individual trials substantiate the effectiveness of comprehensive occupational therapy, foot orthoses, finger splints and wrist working splints, but not of wrist resting splints. Overall, the evidence for the effectiveness of assistive devices and dietary interventions is scanty. SUMMARY: For exercise and physical activity programmes and self-management interventions in RA, research is increasingly directed towards the optimization of their content, intensity, frequency, duration and mode of delivery and effective implementation strategies. A number of studies substantiate the effectiveness of comprehensive occupational therapy, wrist working splints and finger splints. More research into the effectiveness of assistive devices, foot orthoses and dietary interventions is needed. Show less