Galili Naomi, Sechman Eric V, Cerny Jan, Mehdi Murtaza, Mumtaz Muhammad, Westervelt Peter, Maguire Jessica, Raza Azra
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.
Leuk Res. 2007 Jan;31(1):19-26. doi: 10.1016/j.leukres.2006.04.009. Epub 2006 Oct 24.
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.
骨髓增生异常综合征（MDS）是一组造血系统疾病，伴有不同程度的一系到三系血细胞减少和骨髓发育异常。近年来，MDS的治疗取得了很大进展，目前有几种治疗化合物在不同程度上取得了成功应用。这些化合物通常会引起副作用，这使得它们对于治疗处于MDS早期阶段的患者缺乏吸引力。无毒的天然化合物可能为治疗疾病初期的患者提供一种方法。我们进行了一项初步研究，以测试辅酶Q10（coQ10）对低至中2风险疾病的MDS患者的疗效。在29名患者中的7名患者中观察到了多种反应，包括两个三系反应和两个细胞遗传学反应。对预处理骨髓的线粒体DNA（mtDNA）进行测序显示，在3/5无反应者、1/4反应者以及两个对照样本中存在多个突变，其中一些突变导致氨基酸变化。我们得出结论，coQ10可能对一部分MDS患者具有临床益处，但不能根据传统的临床和病理特征或mtDNA突变轻易预先选择反应者。
