Complete remission following clofarabine treatment in refractory juvenile myelomonocytic leukemia.
Juvenile myelomonocytic leukemia (JMML) is the most common myeloproliferative/myelodysplastic disorder seen in children. The treatment of choice, allogeneic stem cell transplantation, provides the only known cure for the disease, but relapse after transplant is common. The authors describe a 5-year-old boy diagnosed at age 34 months with JMML that evolved to acute myeloid leukemia. Initial treatment consisted of fludarabine and cis-retinoic acid therapy, followed by a matched sibling bone marrow transplant. After a relapse, he received a second transplant from the same donor, using peripheral blood stem cells, followed by repeated donor leukocyte infusions. After the second relapse, he received the farnesyltransferase inhibitor R115777 (tipifarnib, Zarnestra), but the leukemia persisted. When bone marrow blasts numbered 60% of the mononuclear cells, he received single-agent clofarabine induction (52 mg/m/d) for 5 days. After three courses, he attained a remission marrow with 5% blasts and disappearance of the 5q- and 9q- cytogenetic abnormalities.
Corey
Seth J
SJ
Section of Pediatric Leukemia/Lymphoma, Division of Pediatrics, University of Texas, M.D. Anderson Cancer Center, Houston, Texas 77030, USA. sjcorey@mdanderson.org
Elopre
Marily
M
Weitman
Steve
S
Rytting
Michael E
ME
Robinson
Lisa J
LJ
Rumelhart
Stephen
S
Goldman
Frederick D
FD
D000227:Adenine Nucleotides; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001087:Arabinonucleosides / Q000627:therapeutic use*; D016026:Bone Marrow Transplantation; D002648:Child; D000077866:Clofarabine; D019008:Drug Resistance, Neoplasm; D006801:Humans; D015477:Leukemia, Myelomonocytic, Chronic / Q000188:drug therapy*; D008297:Male; D009364:Neoplasm Recurrence, Local / Q000188:drug therapy; D012074:Remission Induction; D016896:Treatment Outcome
D000227:Adenine Nucleotides; D000970:Antineoplastic Agents; D001087:Arabinonucleosides; D000077866:Clofarabine