Long-term Benefit With Stem-Cell Transplant in Scleroderma

Published: Nov 21, 2014

By Kate Johnson , Contributing Writer, MedPage Today

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• Note that these studies were published as abstracts and presented at a conference. These data and conclusions should be considered to be preliminary until published in a peer-reviewed journal.

BOSTON -- Patients with rapidly progressing systemic sclerosis (SSc) showed sustained benefits over 5 years after autologous hematopoietic stem-cell transplantation (HSCT), compared with nontransplanted patients, researchers reported here.

"Our study confirms that HSCT induces a good and persistent improvement in cutaneous involvement and stabilization in lung involvement," said lead investigator Eleanora Zaccara, MD, from the UOC Day Hospital of Rheumatology, Gaetano Pini Orthopedic Institute, in Milan, Italy.

"We think that HSCT could represent a new therapeutic opportunity for patients suffering from systemic sclerosis, but the accurate selection of patients represents a crucial therapy decision."

The retrospective study, included 18 patients with diffuse cutaneous SSc who underwent HSCT at Zaccara's hospital from 2003 to 2009.

Median age was 41 years and median disease duration was 24 months, reported the study's senior investigator Nicoletta Del Papa, MD, PhD, from the same hospital, who presented the findings at theannual meeting of the American College of Rheumatology. Thirteen of the group were female.

All patients had rapidly progressive disease with a modified Rodnan Skin Score (mRSS) greater than 14 and a clinical activity score above 3, evaluated according to the European Scleroderma Study Group (ESSG) system.

Patients were excluded if they had severe organ impairment, defined as lung diffusion capacity (DLCO) of less than 40% or a cardiac ejection fraction of less than 45%.

After a median follow-up of 60 months, there was a transplant-related mortality of 5.8%: one patient died from interstitial pneumonia at day 65, and another died from disease progression (fatal cardiac arrhythmia) at 3 years, said Del Papa.

Among the surviving patients there was sustained improvement in skin thickening: from a mean baseline mRSS of 19.8 to 9.3 at 6 months, 6.2 at 1 year and 3.0 at year 5 (P<0.001), she said.

In addition, there was a persistent reduction in disease activity measured by ESSG score, from a mean of 5.3 at baseline, to 2.1 at 6 months, 2.0 at 1 year, and 1.5 at year 5 (P<0.0001).

There was no progressive organ involvement during the 5-year follow-up. The mean DLCO was 65.0% at baseline, 67.2% at year one, and 58.0% at year five, with the mean vital capacity value of 81% at baseline, and 82% and 89.2% at years one and five respectively.

Comparing the transplant patients with 36 age- and sex-matched SSc control patients who had not received HSCT, the study showed that transplant patients had significantly better mRSS (P<0.001), DLCO (P=0.0004), and ESSG scores (P<0.001) at 5 years, as well as significantly improved survival (P=0.0004), she said.

"Our analysis shows that the natural history of the disease is quite different from the history of the transplanted patients in terms of survival and disease progression," noted Zaccara.

However, "a possible bias of our study could be the fact that we compared transplanted patients to this historic cohort where a minority were aggressively treated," added Del Papa.