The edition · Rheumatology
Ten years after stem-cell transplant for systemic sclerosis, 60% remained progression-free
Long-term follow-up of 80 European and Brazilian patients sets a benchmark for cellular therapies. Also: immunomodulation and survival in autoimmune lung fibrosis, rituximab in antiphospholipid syndrome, and a better way to check bisphosphonates are working.
The edition in brief
Long-term follow-up of 80 patients who had autologous haematopoietic stem-cell transplantation for severe progressive systemic sclerosis between 2012 and 2016 found progression-free survival of 81% at 2 years, 72% at 5 and 60% at 10, with 43% in drug-free remission throughout; 6% died of transplant-related causes. In a Madrid registry of 158 patients with autoimmune-associated progressive pulmonary fibrosis, most deaths were from lung disease, and immunomodulatory treatment was associated with lower lung-related mortality (subhazard ratio 0.35), though treatment was not randomised. A single-centre series of 54 patients with antiphospholipid syndrome found rituximab sharply reduced antibody positivity at 6 to 12 months, but titres drifted back, and most recurrent thromboses occurred off anticoagulation or with low INR. In 108 women randomised to oral bisphosphonates, bone turnover markers CTX and PINP identified treatment response in 94% to 96% within three months, while bone density changes at two years were too small to separate responders reliably.
Stem-cell transplant for systemic sclerosis: durable benefit at ten years
Consider early referral of selected patients with severe progressive systemic sclerosis for transplant assessment; benefit can last a decade.
Immunomodulation linked to longer survival in autoimmune progressive pulmonary fibrosis
In autoimmune progressive pulmonary fibrosis, consider continuing immunomodulatory therapy; it was linked to substantially lower lung-related mortality.
Rituximab in antiphospholipid syndrome: antibodies fell, then drifted back
Rituximab lowered antiphospholipid antibodies briefly, but recurrent clots mostly followed lapses in anticoagulation — keep anticoagulation central.
Hydroxychloroquine: dose by weight and screen the retina
Keep hydroxychloroquine at or below 5 mg/kg/day and arrange baseline and annual retinal screening after five years.
Check bisphosphonate response with bone turnover markers at three months
Consider checking CTX or PINP at baseline and three months to confirm an oral bisphosphonate is working, rather than waiting for repeat DXA.
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