BRIGHTON, ENGLAND – Survival from scleroderma is improving, but major organ-based complications are still a significant reason for premature death, according to the results of a long-term outcomes study that were presented at the annual meeting of the British Society for Rheumatology.
The 10-year survival rates were 81% for patients with limited cutaneous systemic sclerosis (SSc) and 70% for patients with diffuse cutaneous SSc (P = .006).
Survival was significantly reduced (39%) in those who developed major heart, lung, or kidney complications, compared with 71% for those who developed no complications at 15 years' follow-up (P less than .001).
"The 10-year survival is impressive when you consider that not so long ago, we had a 5-year morality approaching 50% in the worst cases. So clearly there has been overall progress in managing the disease," commented study investigator Dr. Christopher Denton.
"Although outcomes are improving, we're still dealing with a disease that has very high case-specific mortality," added Dr. Denton, who is a professor of experimental rheumatology at University College London and a consultant rheumatologist at the Royal Free Hospital, London.
"Around 60% of patients actually die as a direct consequence of their scleroderma, and this is mostly from cardiorespiratory complications of the disease," he added.
Almost 400 patients with scleroderma – of whom 252 (63.3%) had limited cutaneous SSc and 146 (36.7%) had diffuse cutaneous SSc – were studied from their diagnosis in 1995 to 1999 (Rheumatology [Oxford] 2011;50 [suppl. 3]:iii129-iii37, abstract 223). The percentage of men with diffuse cutaneous SSc vs. limited cutaneous SSc disease was higher (17.9% vs. 9.9%; P = .037) and the mean age of onset was 50 years for limited cutaneous SSc patients and 46 years for diffuse cutaneous SSc patients (P = .003).
An important point of the study was that the frequency and timing of the major complications of scleroderma have now been defined over a 10- to 15-year period. Complications of scleroderma can be life threatening, and include pulmonary arterial hypertension (PAH), pulmonary fibrosis, renal disease, and cardiac problems.
At the 10-year follow-up, the incidences of PAH in the limited cutaneous SSc and diffuse cutaneous SSc groups were similar (15% vs. 13%), with the earliest cases seen 2 years after diagnosis. Patients who developed this complication later in the course of their disease tended to have higher 5-year survival rates than did those who had PAH earlier (49% vs. 24%), but this did not reach statistical significance.
Clinically significant pulmonary fibrosis was identified in a higher percentage of patients with diffuse than with limited disease at 10 years (45% vs. 21%; P less than .001), with almost half of patients developing lung disease within the first 3 years, and more than three-quarters within the first 5 years.
Patients with diffuse cutaneous SSc were more likely than those with limited cutaneous SSc to experience renal crisis (12% vs. 4%) at 10 years, with the higher rates occurring in the first 3 years (10% vs. 3%). Similarly, cardiac disease was more common in diffuse cutaneous SSc than in limited cutaneous SSc patients early on (7% vs. 1% at 5 years; P less than .001), but remained unchanged at 10 years.
"Whereas the limited or diffuse distinction is very relevant in the first 5 years, after the 5 years it doesn’t really matter which type of scleroderma you’ve got; you have an equally high frequency of complications," Dr. Denton observed in an interview.
He added that this "is a treated cohort that received standard management, so I think it’s very important to start to understand the timing and frequency of the major heart, lung, and kidney complications, so that you can really identify when in the course of the disease you should be particularly vigilant."
Dr. Denton disclosed financial relationships with Actelion, Aspreva, BioVitrum, Digna, Dyax, Encysive, Genzyme, GlaxoSmithKline, Medimmune, Pfizer, and Sanofi-Aventis.