Hematopoietic stem cell transplantation for scleroderma durable after 6 to 11 years
CHICAGO — A long-term follow-up to the Scleroderma: Cyclophosphamide or Transplantation trial demonstrated that the clinical benefits of myeloablative autologous hematopoietic stem cell transplantation were durable 6 to 11 years following initial randomization, according to Keith Sullivan, MD, of Duke University Medical Center.
“The Scleroderma: Cyclophosphamide or Transplantation (SCOT) study compared cyclophosphamide and myeloablative CD34-selected autologous hematopoietic stem cell transplantation,” Sullivan said, addressing attendees at the ACR/ARHP 2018 Annual Meeting. “As you know all too well, scleroderma with internal organ involvement is a life-threatening autoimmune disease in need of more effective treatment. Published earlier this year, the study showed that outcomes were superior with transplantation compared to those with monthly cyclophosphamide.”
To report on survivor status, late effects and outcomes 6 to 11 years after initial entry to the SCOT trial, Sullivan and colleagues in 2017 conducted scripted telephone interviews with the surviving participants and searched public death records. Study endpoints included time to death or organ failure.
Interviews with surviving participants covered current health status, physical functioning, toxicities, disease-modifying antirheumatic drug use and quality of life measures. In addition, participants’ Health Assessment Questionnaire Disability Index and short form health survey (SF-36) answers were compared with their last assessments.
According to Sullivan, of the 75 participants initially randomized in the SCOT trial, seven who had received hematopoietic stem cell transplantation, and 18 treated with cyclophosphamide, have died. Among those who received transplantation, no new deaths were identified during the follow-up study, whereas four new deaths were recorded among the cyclophosphamide group. At 11 years following initial enrollment, Kaplan Meier estimates of overall survival were 80% for transplantation compared with 52% for cyclophosphamide in the intention-to-treat population. In the per-protocol population, which received transplantation or nine or more doses of cyclophosphamide, overall survival estimates were 88% for transplantation and 53% for cyclophosphamide.
Among the 25 participants who received transplantation, and the 18 treated with cyclophosphamide, in the follow-up study, physical functioning and weight gain were improved following transplantation. Organ failure was recorded in two participants who received transplantation, compared with six in the cyclophosphamide group. In addition, performance status was significantly improved following transplantation. A total of two patients who received transplantation were taking DMARDs, compared with seven in the cyclophosphamide group, with 92% and 61% of the two respective groups remaining DMARD-free (P = .01).
“This is worth thinking about,” Sullivan said. “Here is a frustrating autoimmune disease wherein patients have improved and have been off DMARDs for more than a decade. Laboratory studies presented at this meeting further define the mechanisms of treatment response. Early referral for transplant consultation before organ failure will allow shared decision making with patients regarding choice of treatment.” – by Jason Laday
Disclosure: Sullivan reports no relevant financial disclosures.
Sullivan K. Abstract 1820. Presented at ACR/ARHP Annual Meeting, Oct. 20-24, 2018; Chicago.