Abstract
Sezary syndrome (SS) and advanced tumor-stage mycosis fungoides (MF) have an aggressive clinical course, ultimately leading to a very short survival. Because patients are often elderly and show poor clinical conditions, conventional allogeneic hematopoietic stem cell transplantation is associated with a undesirable high TRM; therefore, allogeneic reduced-intensity conditioning (RIC) HSCT represent an attractive and worth-investigating strategy of cure in this disease setting. In our Institutions, from September 2000 to May 2006, fifteen patients with advanced mycosis fungoides or Sezary Sindrome underwent RIC allogeneic hematopoietic stem cell transplantation either from HLA-identical sibling, or from HLA-matched unrelated donor, or from unrelated cord blood. Conditioning regimens for patients with HLA-identical sibling included fludarabine/cyclophosphamide/TBI200 up to 2001, then pentostatin/TBI200. Patients who underwent unrelated transplants were treated with melphalan/CP1H/Fludarabine/TBI200 combination. All patients (10 males and 5 females) had a clinical diagnosis of MF (10 pts) or SS (5 pts) confirmed by histopathology, immunohistochemistry and molecular biology. Median age was 48 years (range 38–65). Median time from diagnosis to transplant was 48 months (range 13 to 252). Disease stage for all patients was from IIIB to IVB. GVHD prophylaxis included oral Cy-A from day 0 to day +100 and oral MMF from day 0 to day +27, then tapered within 2 weeks. The source of donor stem cells was peripheral blood and bone marrow for siblings and MUD, respectively. A complete clinical remission was achieved in 10 patients, whereas 2 patients obtained a VGPR. Five patients died from various complications (3 with progressive disease, 1 in CR e 1 in PR) within a period ranging from 33 days to 8 months after transplant. A full donor chimerism was achieved within 6 months in 8 out of 10 evaluable patients, whereas no engraftemnt was observed in 2 patients (1 MUD and 1 UCB). 5 patients experienced acute GVHD of grade I–II, while limited chronic GVHD occurred in 7 patients. With a median follow-up of 38 months (range 2–70), ten patients were alive with an estimated overall survival greater than 60% at five years (Figure 1), whereas 7 patients were alive and disease-free at the last follow-up. Based on these highly encouraging results, we conclude that RIC allogeneic HSCT represents a feasible and effective treatment for advanced mycosis fungoides and Sezary Syndrome. Further studies in larger patient populations are warranted to confirm the curative potential of this strategy.
Overall Survival in MF/SS following RIC allo-HSCT Overall Survival in MF/SS following RIC allo-HSCT
Safety and Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Mycosis Fungoides and Sezary Syndrome: A 7-year Tertiary Center Analysis