Non-Myeloablative Allogeneic HSCT in High Risk Patients with Myelofibrosis. Session Type: Oral Session

Damiano Rondelli, Gianni Barosi, Andrea Bacigalupo, Joseph T. Prchal, Uday Popat, Emilio P. Alessandrino, Jerry L. Spivak, Ronald Hoffman, Steven Fruchtman

Author address: 

University of Illinois at Chicago, Chicago, IL, USA; IRCCS Policlinico San Matteo, Pavia, Italy; Ospedale San Martino, Genova, Italy; Baylor College of Medicine, Houston, TX, USA; Johns Hopkins University, Baltimore, MD, USA; Mount Sinai School of Me


Myelofibrosis is a rare chronic myeloproliferative disorder without curative treatment. The main therapeutic option remains transfusion support, with significant morbidity due to massive organomegaly. For patients with high risk Lille scores (Hb30,000) the median survival is less than 2 years. Fully ablative allogeneic conditioning results in a 25% peritransplant mortality, with few long term DFS in this typically elderly group. We report on 20 patients, median age 54 yrs (range:27-68), who received a non-myeloablative allogeneic HSC transplantation from HLA-matched related (n=18) or unrelated (n=1), or 1Ag-mismatched related donor (n=1). At the time of transplant median values of Hb, Plt, and WBC were: 8.1 g/dL (range: 6.2-10.7); 70 x 109/L (range: 8-278) and 5.0 x 109/L (range: 0.7-28.7), respectively. Marked splenomegaly was present in 15 pts;, in the remaining 5 , 3 were splenectomized. Eighteen of 20 pts had grade III/IV marrow fibrosis and 9 pts were RBC and 3 PLT transfusion-dependent. Reduced intensity conditioning (RIC) regimens included low doses TBI (200cGy) and Fludara (n=5), Fludara/Melphalan (n=6), Thiotepa/EDX (n=8) and Thiotepa/Fludara (n=1). Engraftment of ANC >500 and Plt >20K occurred within days 12-18 and 16-77, respectively, in all pts. Chimerism analysis on d30 showed >90% donor cells in 18/20, while the remaining 2 (70% and 56%) achieved 100% after DLI. Acute GVHD grade II-IV was observed in 5 patients and chronic GVHD in 8 of 16 evaluable pts. Day 100 TRM was 0%. Eighteen of 20 pts (90%) are alive at 18 months median follow-up. Two deaths were due to aGVHD and aGVHD + aspergillosis (post DLI). Marrow fibrosis was reduced to grade 1 in the majority of the patients and median CBC values are: Hb 10.0 g/dL (range: 8.1-15.5), Plt 110 x 109/L (range: 17-340), and WBC 6.4 x 109/L (range: 0.7-12.0). Splenomegaly was dramatically reduced in all patients. Non-myeloablative allogeneic HSC transplantation can be safely performed in MMF patients, with 1) low TRM 2) eradication of marrow fibrosis and massive organomegaly and 3) restoration of more normal hematopoiesis. These provocative outcomes will prompt randomized trials in MMF, including allogeneic transplantation. Abstract #695 appears in Blood, Volume 102, issue 11, November 16, 2003

abstract No: 


Full conference title: 

American Society of Hematology 45th Annual Meeting
    • ASH 45th (2003)