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TOPIC: New Systematic Scleroderma Study Shows Good Results

New Systematic Scleroderma Study Shows Good Results 04 Jan 2018 13:22 #10850

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www.nejm.org/doi/full/10.1056/NEJMoa1703327

BACKGROUND
Despite current therapies, diffuse cutaneous systemic sclerosis (scleroderma) often has a devastating outcome. We compared myeloablative CD34+ selected autologous hematopoietic stem-cell transplantation with immunosuppression by means of 12 monthly infusions of cyclophosphamide in patients with scleroderma.

METHODS
We randomly assigned adults (18 to 69 years of age) with severe scleroderma to undergo myeloablative autologous stem-cell transplantation (36 participants) or to receive cyclophosphamide (39 participants). The primary end point was a global rank composite score comparing participants with each other on the basis of a hierarchy of disease features assessed at 54 months: death, event-free survival (survival without respiratory, renal, or cardiac failure), forced vital capacity, the score on the Disability Index of the Health Assessment Questionnaire, and the modified Rodnan skin score.

RESULTS
In the intention-to-treat population, global rank composite scores at 54 months showed the superiority of transplantation (67% of 1404 pairwise comparisons favored transplantation and 33% favored cyclophosphamide, P=0.01). In the per-protocol population (participants who received a transplant or completed ≥9 doses of cyclophosphamide), the rate of event-free survival at 54 months was 79% in the transplantation group and 50% in the cyclophosphamide group (P=0.02). At 72 months, Kaplan–Meier estimates of event-free survival (74% vs. 47%) and overall survival (86% vs. 51%) also favored transplantation (P=0.03 and 0.02, respectively). A total of 9% of the participants in the transplantation group had initiated disease-modifying antirheumatic drugs (DMARDs) by 54 months, as compared with 44% of those in the cyclophosphamide group (P=0.001). Treatment-related mortality in the transplantation group was 3% at 54 months and 6% at 72 months, as compared with 0% in the cyclophosphamide group.

CONCLUSIONS
Myeloablative autologous hematopoietic stem-cell transplantation achieved long-term benefits in patients with scleroderma, including improved event-free and overall survival, at a cost of increased expected toxicity. Rates of treatment-related death and post-transplantation use of DMARDs were lower than those in previous reports of nonmyeloablative transplantation. (Funded by the National Institute of Allergy and Infectious Diseases and the National Institutes of Health; ClinicalTrials.gov number, NCT00114530.)

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New Systematic Scleroderma Study Shows Good Results 04 Jan 2018 14:48 #10851

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Glad someone can run a trial !!!
Progression free survival and overall survival HUGE with the FDA.
Stat Sig across the board.
Maybe someday gets lucky and can run a late stage trial with a satisfactory conclusion....but this shows Cytori still far from prime time.

WST...how is the snow in your area ?

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New Systematic Scleroderma Study Shows Good Results 04 Jan 2018 15:13 #10852

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Hedge

This combo multicenter trail was completed long time ago and the study results are known a long time ago.

For FDA to approve the combination (both are experimental), the first step to get the stem cells approval and then combination. FDA would not approve such a combination in the first cut. Cytori's Habeo (or any other cell therapies) may be marginal statisitcally, but the combination would be powerful It would be a waste time to keep doing STAR trial - the FDA should allow limited claims for Habeo and this would allow CYTX and the medical community to do the combination under a phase 4 setting or a new phase 3 trial.

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New Systematic Scleroderma Study Shows Good Results 04 Jan 2018 15:22 #10853

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franshei wrote: Hedge

This combo multicenter trail was completed long time ago and the study results are known a long time ago.

For FDA to approve the combination (both are experimental), the first step to get the stem cells approval and then combination. FDA would not approve such a combination in the first cut. Cytori's Habeo (or any other cell therapies) may be marginal statisitcally, but the combination would be powerful It would be a waste time to keep doing STAR trial - the FDA should allow limited claims for Habeo and this would allow CYTX and the medical community to do the combination under a phase 4 setting or a new phase 3 trial.


Franshei, thanks for keeping it honest - b/t/w, you did not respond to my earlier question?

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New Systematic Scleroderma Study Shows Good Results 04 Jan 2018 17:45 #10855

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franshei
Doesn't change one thing in my post...LOL
Also, if I am remembering correctly you said somewhere in the past stat sig doesn't matter...when it surely does and survival is the gold standard for the FDA.
Others are producing meaningful data...that's the rub !
As far as a phase 4....we are still waiting to hear...still it did make for a nice carrot to buy the RO this round. You know my feelings on those chances.

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New Systematic Scleroderma Study Shows Good Results 04 Jan 2018 21:11 #10861

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Hey Hedge. I'd say about a foot and a half but drifts up to 5 feet!

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