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The Medical & Scientific Advisory Board of dure which would exclude many of our

the Scleroderma Foundation released the current patients.
This study is an extremely valuable
following statement on January 8, 2018 contribution to the treatment of sclero-
derma. However, there are numerous
unanswered questions, some of which
The results of the National Institutes only two patients died from progression are being addressed in follow-up studies,
of Health (NIH) supported multi-center of their scleroderma, compared to 11 including how long the improvement lasts
SCOT (Scleroderma: Cyclophosphamide patients treated with Cytoxan indicating (is it in fact life-long or will the disease
or Transplantation) trial were published that the stem cell group had a better eventually relapse), which patients are the
in the January 4, 2018 issue of the New treatment response than the Cytoxan best candidates for this strenuous proce-
England Journal of Medicine. group. However, this needs to be weighed dure, is there a better preparative regimen
against the fact that two patients died in (combination of chemotherapy with/
DESCRIPTION OF THE TRIAL: the stem cell group due to transplant-re- without radiation) that would provide the
The SCOT trial compared the safety lated complications, whereas no patient same results but with fewer side effects?
and benefit of two different immunomod- in the Cytoxan group died from the This treatment is clearly not recom-
ulatory treatments in patients with early treatment. mended for all scleroderma patients. Many
and severe scleroderma. Patients recruited patients show a good response to current
from 26 clinical research sites in North COMPLICATIONS OF THE medical therapies, or show little progres-
America were randomized to either high- TREATMENT: sion of their disease over time. Therefore,
dose chemotherapy followed by hemato- There was a concern that infections it will be important to develop and
poietic stem cell transplant (HSCT) or 12 might be more frequent in one group validate guidelines for identifying those
monthly IV cyclophosphamide (Cytoxan) versus the other but this was not the case. patients who are at high risk for disease
treatments. The stem cells came from the Infections as complications of treatment progression or who have been shown to
patients themselves, not from donors. were seen equally in both groups, but be non-responsive to current treatments.
It is important to point out that the varicella zoster (shingles) infections were In addition, we recommend that patients
treatment procedure in the stem cell more common in the transplant group. for whom stem cell transplantation is
transplant group was composed of two considered a reasonable treatment option
parts: first the patients received high- CONCLUSIONS: be referred for the procedure to expert
dose chemotherapy with irradiation to These results are broadly consistent centers where appropriate evaluative
effectively destroy the patient’s abnormal with favorable outcomes reported from protocols, multi-speciality care - including
immune system, and second, the stem two previous trials in Europe and the US, scleroderma experts and physicians with
cells (removed from the patients prior to and provide support for the use of au- substantial experience with the procedure
chemotherapy) were infused to recon- tologous stem cell transplant therapy for and all of its complications - is available.
stitute the immune system with new or some severe scleroderma patients with The cost of stem cell transplant therapy,
“naive” immune cells that would likely kidney or lung involvement. which can be as high as $150,000, will
not contribute to scleroderma. Thus, the It is always important to weigh advan- likely be an impediment, but it is hoped
therapeutic benefit seen in the stem cell tages versus disadvantages, that is, benefit that the results of the SCOT study will
arm comes from the chemotherapy not versus risk, in any treatment decision par- lead insurers/payors to cover such therapy
from the stem cells themselves. ticularly one with the potential for serious in appropriate situations.
infections and (admittedly uncommon)
RESULTS OF THE TRIAL: treatment-related deaths.
Seventy-five scleroderma patients The patients selected for the study
were enrolled in the study. Each patient had to have adequate kidney and lung
had severe skin disease along with lung function to be able to tolerate the proce-
or kidney involvement. 39 patients were
randomized to treatment with monthly IV
Cytoxan, and 36 patients were random-
ized to receive the high-dose chemother-
apy, irradiation and stem cell transplant.
At an average of four and a half years
of follow-up, patients who received the
chemotherapy, irradiation and stem cell Authored by John Varga, M.D., Maureen D. Mayes, M.D., Virginia D. Steen, M.D., Richard Silver,
transplant had a lower death rate from M.D., and Lorinda Chung, M.D., on behalf of the Scleroderma Foundation’s Medical & Scientific
scleroderma-related causes than pa- Advisory Board. Scleroderma Foundation. 300 Rosewood Drive, Suite 105, Danvers, MA 01923,
tients who received the 12 months of IV www.scleroderma.org, SFinfo@scleroderma.org, 800-722-4673
Cytoxan. In the stem cell-treated group,

38 INSPIRE HEALTH January § February