Clinical response of myelodysplastic syndromes patients to treatment with coenzyme Q10.
The myelodysplastic syndromes (MDS) are a collection of hematopoietic disorders with varying degrees of mono- to trilineage cytopenias and bone marrow dysplasia. In recent years much progress has been made in the treatment of MDS and there are now several therapeutic compounds used with varying levels of success. These compounds typically cause side effects that make them unattractive for treatment of patients in the early stages of MDS. Naturally occurring compounds that are not toxic may provide a means to treat patients in the initial stages of disease. We conducted a pilot study to test the efficacy of coenzyme Q10 (coQ10) in MDS patients with low to intermediate-2 risk disease. A variety of responses were observed in 7 of 29 patients including two trilineage and two cytogenetic responses. Sequencing mitochondrial DNA (mtDNA) from pretreatment bone marrows showed multiple mutations, some resulting in amino acid changes, in 3/5 nonresponders, 1/4 responders and in two control samples. We conclude that coQ10 may be of clinical benefit in a subset of MDS patients, but responders cannot be easily pre-selected on the basis of either the conventional clinical and pathologic characteristics or mtDNA mutations.
Galili
Naomi
N
The Radhey Khanna MDS Center for Research, Division of Hematology, University of Massachusetts Medical Center, 364 Plantation Street, LRB 208, Worcester, MA 01605, United States.
Sechman
Eric V
EV
Cerny
Jan
J
Mehdi
Murtaza
M
Mumtaz
Muhammad
M
Westervelt
Peter
P
Maguire
Jessica
J
Raza
Azra
A
D000368:Aged; D000369:Aged, 80 and over; D001483:Base Sequence; D001854:Bone Marrow Cells / Q000737:chemistry / Q000473:pathology; D003067:Coenzymes; D004272:DNA, Mitochondrial / Q000235:genetics / Q000302:isolation & purification; D005260:Female; D006801:Humans; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors / Q000627:therapeutic use; D007963:Leukocytes, Mononuclear / Q000737:chemistry / Q000473:pathology; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics / Q000503:physiopathology; D013921:Thrombocytopenia / Q000209:etiology / Q000235:genetics / Q000517:prevention & control*; D014451:Ubiquinone / Q000031:analogs & derivatives* / Q000627:therapeutic use
D003067:Coenzymes; D004272:DNA, Mitochondrial; D019161:Hydroxymethylglutaryl-CoA Reductase Inhibitors; D014451:Ubiquinone; C024989:coenzyme Q10