title: Efficacy of myeloablative allogeneic hematopoietic stem cell transplantation in adult patients with MLL-ELL-positive acute myeloid leukemia.
abstract: Acute myeloid leukemia (AML) with mixed lineage leukemia-eleven-nineteen lysine-rich leukemia (MLL-ELL) is a rare subtype of MLL-rearranged AML. The outcome of allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with this disease remains unknown. In the present study, we retrospectively investigated the efficacy of allo-HSCT in eight adult MLL-ELL-positive AML patients. Although all eight patients achieved first complete remission (CR1), three (37.5 %) patients experienced relapse after induction therapy. Five (62.5 %) patients underwent allo-HSCT during CR1, whereas two (25.0 %) underwent allo-HSCT during disease relapse, and one (12.5 %) during CR2. All three patients who received allo-HSCT beyond CR1 died due to AML progression after allo-HSCT. Of the five patients who received allo-HSCT during CR1, three (60.0 %) remained alive at study conclusion. The overall survival rate at five years was 50.0 %. Intriguingly, clonally expanded non-leukemic cells expressing MLL-ELL during consolidation therapy were found to be eradicated after allo-HSCT during the monitoring of minimal residual disease in one patient; this indicates that allo-HSCT is efficacious for eliminating pre-leukemic cells resistant to chemotherapy. In conclusion, allo-HSCT soon after CR1 represents a promising therapeutic option for adult AML patients with MLL-ELL, although the outcome of allo-HSCT for patients beyond CR1 was dismal.
authors.affiliation: Department of Hematology, Chiba University Hospital, 1-8-1 Inohana, Chuo-ku, Chiba, 260-8670, Japan.
authors.forename: Tomoya
authors.initials: T
authors.lastname: Muto
authors.forename: Masahiro
authors.initials: M
authors.lastname: Takeuchi
authors.forename: Atsuko
authors.initials: A
authors.lastname: Yamazaki
authors.forename: Yasumasa
authors.initials: Y
authors.lastname: Sugita
authors.forename: Shokichi
authors.initials: S
authors.lastname: Tsukamoto
authors.forename: Shio
authors.initials: S
authors.lastname: Sakai
authors.forename: Yusuke
authors.initials: Y
authors.lastname: Takeda
authors.forename: Naoya
authors.initials: N
authors.lastname: Mimura
authors.forename: Chikako
authors.initials: C
authors.lastname: Ohwada
authors.forename: Emiko
authors.initials: E
authors.lastname: Sakaida
authors.forename: Nobuyuki
authors.initials: N
authors.lastname: Aotsuka
authors.forename: Tohru
authors.initials: T
authors.lastname: Iseki
authors.forename: Chiaki
authors.initials: C
authors.lastname: Nakaseko
mesh_terms: D000293:Adolescent; D000328:Adult; D019457:Chromosome Breakage; D018450:Disease Progression; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000235:genetics* / Q000401:mortality / Q000628:therapy*; D008297:Male; D051788:Myeloid-Lymphoid Leukemia Protein / Q000235:genetics*; D018365:Neoplasm, Residual / Q000175:diagnosis / Q000235:genetics; D015514:Oncogene Proteins, Fusion / Q000235:genetics*; D020133:Reverse Transcriptase Polymerase Chain Reaction; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: C472772:MLL-ELL fusion protein, human; D015514:Oncogene Proteins, Fusion; D051788:Myeloid-Lymphoid Leukemia Protein