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Mycophenolate mofetil as salvage treatment for steroid-refractory chronic graft versus-host disease in children



Mycophenolate mofetil as salvage treatment for steroid-refractory chronic graft versus-host disease in children



Blood 98(11 Part 1): 398a, November 16



Clinically extensive chronic GVHD occurs in about 40-50% of patients more than 3 months (mos.) after allogeneic transplant (BMT). Most patients (pts.) with chronic GVHD are treated with prednisone (PDN) and cyclosporin (CSP). Despite this therapy, only 30% of pts. achieve complete response (CR) and discontinue therapy during the first 3 years post-BMT. To determine whether addition of a third agent would potentially improve the CR rate, we evaluated the impact of adding MMF to the PDN and CSP therapy for 26 consecutive children, 1-9 (median 7) yrs. of age, who had clinically extensive chronic GVHD that progressed on therapy with PDN and CSP. The 26 pts. had BMT for ALL/NHL (n=9), AML/MDS/CML (n=16) and SCIDS (n=1). Stem cell donors were HLA-matched related (n=7), mismatched related (n=3), matched unrelated (n=9) and mismatched unrelated (n=7). Sources of stem cells were bone marrow (n=22), peripheral blood (n=3) and cord blood (n=1). MMF 20mg/kg bid was added at a median of 14 mos. (range 3-28) post-BMT. The median duration of treatment with MMF was 14 mos. (range 9-35). Evaluations of chronic GVHD status were performed every 8-12 mos. Patients were considered successfully treated when there was resolution of clinical, histologic and biochemical evidence of chronic GVHD and all immunosuppressive therapy successfully discontinued. Results of these evaluations are given. Toxicity to MMF occurred in only 1 (4%) patient who had transient leucopenia not requiring discontinuation of MMF. Overall, GVHD chronic CR rate was 65% (n=17). MMF was successfully discontinued after 9-12 mos. in 5, after 12-18 mos. in 4, after 18-24 mos. in 3, and 24-36 mos. in 5 pts. In addition, 3 (12%) of the 26 pts. are currently tapering off treatment as their chronic GVHD is quiescent. Six (23%) failed therapy, with progressive chronic GVHD, and 4 of these 6 required additional therapy. Twenty two (85%) of the 26 pts survive disease-free for a median of 4.7 yrs. (range 2.9-6.8) post-BMT. Four (15%) pts. died, 2 (8%) due to progressive chronic GVHD while on MMF and 2 (8%) of relapse of their primary malignancy. These data suggest an excellent CR and PR rate of 77% for children with progressive chronic GVHD when MMF is added to PDN and CSP therapy. A prospective randomized study of newly diagnosed chronic GVHD patients is needed to determine whether this approach can improve the initial chronic GVHD treatment response rate.

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Accession: 035360099

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