The role of cytotoxic therapy with hematopoietic stem cell transplantation in the therapy of myelodysplastic syndromes: an evidence-based review.

Clinical research examining the role of hematopoietic stem cell transplantation (SCT) in the therapy of myelodysplastic syndromes (MDS) in adults is presented and critically evaluated in this systematic evidence-based review. Specific criteria were used for searching the published literature and for grading the quality and strength of the evidence and the strength of the treatment recommendations. Treatment recommendations based on the evidence are presented in Table 3, and were reached unanimously by a panel of MDS experts. The identified priority areas of needed future research in MDS include: (1) the benefit of using alternative donor sources (eg, cord blood; haploidentical family donors) for patients without matched sibling or unrelated donors; (2) the role and appropriate timing of allogeneic SCT in combination with hypomethylating and immunomodulatory treatment regimens; (3) randomized trials comparing the safety and efficacy of various novel agents for treating MDS; and (4) the influence of the various MDS treatment modalities on patient-reported quality-of-life outcomes.

Common troublesome symptoms and their impact on quality of life in patients with myelodysplastic syndromes (MDS): results of a large internet-based survey.

Despite the clinical importance of health-related quality of life (QOL) in patients suffering from myelodysplastic syndromes (MDS), few data exist on the prevalence of key MDS-associated symptoms, or the correlation of those symptoms with specific disease features such as hemoglobin level. In order to better understand the burden of disease-associated symptoms in patients with MDS, we designed a 120-question Internet-based survey of QOL appropriate for patients with MDS, incorporating validated QOL measurement instruments and questions about specific therapies. The 359 survey respondents were typical of MDS patients in terms of demographics, blood counts, and disease subtype. Patients reported high levels of excessive fatigue and poor scores on QOL assessments such as the Functional Assessment of Cancer Therapy-Anemia (FACT-An) and the Brief Fatigue Inventory (BFI). Patients' debilitating fatigue correlated poorly with hemoglobin level, and fatigue was associated with significant impairment of both health-related QOL and ability to work or participate in desired activities. Within the limitations of self-reported data, these results provide a benchmark for future interventions to improve QOL in patients with MDS.