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SAT0471 Autologous Hematopoietic Stem Cell Transplantation in Rapidly Progressive Systemic Sclerosis is More Effective Than Conventional Therapies in Inducing Disease Remission and Prologing Survival
  1. N. Del Papa1,
  2. E. Zaccara1,
  3. W. Maglione1,
  4. D. Sambataro1,
  5. G. Sambataro1,
  6. R. Andracco1,
  7. G. Saporiti2,
  8. R. Giordano3,
  9. C. Vitali4,
  10. A. Cortelezzi2,
  11. F. Onida2
  1. 1UOC DH Reumatologia, Ist. G. Pini
  2. 2Hematology-BMT Unit
  3. 3F. Calori Cell Factory, Policlinico-Univ. di Milano, Milano
  4. 4Reumatologia, Ist. S. Giuseppe, Como, Italy


Background Autologous haematopoietic stem cell transplantation (AHSCT) has shown to be an effective therapeutic option to prolong survival of patients (pts) suffering from rapidly progressive diffuse cutaneous systemic sclerosis (rp-dcSSc).

Objectives This study was aimed at retrospectively comparing the disease outcome of pts with rp-dcSSc treated with AHSCT to that of age-, and sex-matched group of clinically similar pts selected from our cohort and treated with conventional therapies.

Methods Eighteen pts (5 M, 13F; median age 40, 20-62 yrs) that underwent AHSCT were compared to 36 age- and sex-matched pts (10 M, 26 F; median age 44, 19-62 yrs), all of them suffering from rp-dcSSc. At the starting evaluation point the two groups were also exactly comparable for disease-duration and clinical findings, in term of skin involvement (mRSS), DLCO as % of predicted value and disease activity (ESSG scoring system). AHSCT was performed by mobilisation with cyclophosphamide (CTX) and G-CSF, selection of CD34+ cells and conditioning regimen with CTX and rabbit ATG. Twenty-five pts in the control group received 6 monthly pulses of intravenous CTX (750mg/m2), 11 received steroids, DMARDS (Methotrexate and Azathioprine) and vasoactive therapies (noCTX). The clinical course in AHSCT and control groups was evaluated from time 0 to 5 years by using Kaplan Meyer survival curves, computing the Hazard Ratio (HR) and Chi square distribution.

Results Results of the survival curve analysis of different groups, expressed as HR (95%CI) are reported in the table.

Table 1

Conclusions This retrospective study confirms that AHSCT approach is more effective to induce a longer survival (p=0.0004), and shows that is also able to more rapidly reduce the mRss (p<0.0001), disease activity (p<0.0001), and preserve lung function (p=0.0004), with respect to conventional therapies in rp-dcSSc.

Disclosure of Interest None declared

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