title: Induction of a hematological and cytogenetic remission in a patient with a myelodysplastic syndrome secondary to Fanconi's anemia employing the S-HAM regimen.
abstract: We report on a patient with Fanconi's anemia (FA) who developed a myelodysplastic syndrome (RAEB-T) with complex karyotypic abnormalities (trp 1q23q42, monosomy 20, trisomy 13) at the age of 28. The patient achieved a complete hematological and cytogenetic remission after treatment with sequential high-dose cytosine arabinoside/mitoxantrone followed by G-CSF (5 micrograms/kg). Bone marrow hypoplasia was prolonged with 38 days of granulocytopenia < 500/microliters and 62 days of platelet transfusion dependency. Nonhematological toxicity did not exceed that of patients without underlying FA. Remission duration was 7 months. This observation shows the feasibility of high-dose Ara C treatment in patients with FA and MDS. Although hematopoiesis remained clonal in remission, the suppression of the cytogenetically abnormal clones transiently reversed the antecedent long-lasting pancytopenia.
authors.affiliation: Department of Hematology/Oncology, Georg-August University, Göttingen, Germany.
authors.forename: W
authors.initials: W
authors.lastname: Verbeek
authors.forename: D
authors.initials: D
authors.lastname: Haase
authors.forename: C
authors.initials: C
authors.lastname: Schoch
authors.forename: W
authors.initials: W
authors.lastname: Hiddemann
authors.forename: B
authors.initials: B
authors.lastname: Wörmann
mesh_terms: D000328:Adult; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000627:therapeutic use*; D002869:Chromosome Aberrations; D003561:Cytarabine / Q000008:administration & dosage; D005199:Fanconi Anemia / Q000150:complications*; D005260:Female; D006801:Humans; D007621:Karyotyping; D008942:Mitoxantrone / Q000008:administration & dosage; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000209:etiology / Q000235:genetics*; D012074:Remission Induction
chemical_list: D003561:Cytarabine; D008942:Mitoxantrone