title: Cytosine arabinoside and mitoxantrone followed by second allogeneic transplant for the treatment of children with refractory juvenile myelomonocytic leukemia.
abstract: Hematopoietic stem cell transplantation (HSCT) remains the only curative option for most patients with juvenile myelomonocytic leukemia (JMML). However, persistent disease and relapse rates after transplant range from 26% to 58%. We report the successful use of second HSCT after preparation with mitoxantrone and cytosine arabinoside (Ara-C) for patients with refractory or recurrent disease. Between 1993 and 2006, 5 children who underwent HSCT at our institution as initial therapy for JMML had persistent disease or relapsed. Pre-HSCT conditioning varied and donors were either HLA-matched siblings (n=2) or matched unrelated donors (n=3). After initial HSCT, they subsequently received high-dose Ara-C (3 g/m IV) every 12 hours on days -8 through -3 and mitoxantrone (10 mg/m/d IV) on days -8, -7, -6 followed by second HSCT from their original donors. All 5 patients are alive at 88, 179, 199, 234, and 246 months with no evidence of JMML, no significant toxicity, and 100% donor chimera as determined by PCR short-tandem repeat analysis. Our experience supports second transplant utilizing high-dose Ara-C and mitoxantrone in children with JMML who do not respond or relapse after first transplant.
authors: *Division of Pediatric Hematology/Oncology and Bone Marrow Transplantation †Department of Pathology and Microbiology ‡Division of Clinical and Human Genetics, Munroe-Meyer Institute, The University of Nebraska Medical Center, Omaha, NE.
authors: Sachit A
authors: SA
authors: Patel
authors: Don W
authors: DW
authors: Coulter
authors: Alfred C
authors: AC
authors: Grovas
authors: Bruce G
authors: BG
authors: Gordon
authors: James L
authors: JL
authors: Harper
authors: Phyllis I
authors: PI
authors: Warkentin
authors: James L
authors: JL
authors: Wisecarver
authors: Warren G
authors: WG
authors: Sanger
authors: Peter F
authors: PF
authors: Coccia
mesh_terms: D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D003561:Cytarabine / Q000008:administration & dosage*; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D054429:Leukemia, Myelomonocytic, Juvenile / Q000188:drug therapy* / Q000628:therapy*; D008297:Male; D008942:Mitoxantrone / Q000008:administration & dosage*; D012008:Recurrence; D019233:Retreatment; D014019:Tissue Donors; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods; D014184:Transplantation, Homologous
chemical_list: D000964:Antimetabolites, Antineoplastic; D003561:Cytarabine; D008942:Mitoxantrone