British journal of haematology
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Randomized Controlled Trial Multicenter Study
Exercise capacity and haemodynamics in patients with sickle cell disease with pulmonary hypertension treated with bosentan: results of the ASSET studies.
Doppler-defined pulmonary hypertension (PH) in sickle cell disease (SCD) is associated with 40% mortality at 40 months. To assess the effect of bosentan in SCD-PH, two randomized, double-blind, placebo-controlled, 16-week studies were initiated. Safety concerns are particularly relevant in SCD due to comorbid conditions. ⋯ Standard-dose bosentan appears to be well tolerated. Further investigation is warranted. Clinicaltrials.gov registration numbers NCT00310830, NCT00313196, NCT00360087.
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Randomized Controlled Trial Multicenter Study
Abnormalities of chromosome 1p/q are highly associated with chromosome 13/13q deletions and are an adverse prognostic factor for the outcome of high-dose chemotherapy in patients with multiple myeloma.
The prognostic value of chromosomal abnormalities was studied in untreated multiple myeloma patients who were registered into a prospective randomised multicentre phase 3 study for intensified treatment (HOVON24). A total of 453 patients aged less than 66 years with stage II and III A/B disease were registered in the clinical study. Cytogenetic analysis was introduced as a standard diagnostic assay in 1998. ⋯ Patients with karyotypic abnormalities had a significantly shorter overall survival (OS) than patients with normal karyotypes. Complex abnormalities, hypodiploidy, chromosome 1p abnormalities, chromosome 1q abnormalities, and chromosome 13 abnormalities were associated with inferior OS on univariate analysis, as well as after adjustment for other prognostic factors. In conclusion, chromosome 13 abnormalities and chromosome 1p and/or 1q abnormalities were highly associated, and are risk factors for poor outcome after intensive therapy in multiple myeloma.
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Randomized Controlled Trial
A randomised control trial of patient self-management of oral anticoagulation compared with patient self-testing.
Several studies suggest that patient self-management (PSM) may improve the quality of oral anticoagulation therapy as measured by time spent within the international normalised ratio (INR) target range. We performed a prospective randomised control trial to determine whether the improvement in quality of treatment afforded by PSM is greater than that achieved by patient self-testing (PST) alone. A total of 104 of 800 eligible patients aged 22-88 years (median = 59.8), attending our hospital anticoagulant clinic and receiving long-term warfarin for >8 months agreed to participate. ⋯ Both groups combined showed a significant improvement over the previous 6 months (71.0% vs. 62.5%; P = 0.04). Changes in time within the therapeutic range in individual patients (+5.86) also showed a significant difference. The quality of warfarin control in both PST and PSM may be superior to that achieved by conventional management in a specialised hospital anticoagulation clinic.
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Randomized Controlled Trial Comparative Study
Randomised comparison of two B-cell purging protocols for patients with B-cell non-Hodgkin lymphoma: in vivo purging with rituximab versus ex vivo purging with CliniMACS CD34 cell enrichment device.
We investigated the feasibility, safety and efficacy of two B-cell purging methods in patients with CD20+ non-Hodgkin lymphoma (NHL) receiving autologous stsem cell transplantation. Myeloid and immune recoveries between the methods were compared. Twenty-seven patients were randomised to either in vivo purging with rituximab or ex vivo purging by CD34+ cell selection. ⋯ When compared with 53 unpurged patients, all 27 purged patients had improved 3-year probabilities of overall survival (89% vs. 70%, P = 0.014) and a trend for improved EFS (78% vs. 57%, P = 0.075). In conclusion, although both purging methods were feasible and safe, rituximab purging was superior as it did not impair CD34+ cell mobilisation and was associated with faster myeloid recovery. Further studies are needed to determine whether rituximab purging is more effective than the use of unpurged autografts.
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Randomized Controlled Trial Comparative Study Clinical Trial
Superior mobilisation of haematopoietic progenitor cells with glycosylated G-CSF in male but not female unrelated stem cell donors.
Granulocyte colony-stimulating factor (G-CSF) effectively mobilises haematopoietic stem cells to the peripheral blood. It is unclear whether the mobilisation of stem cells with lenograstim (glycosylated G-CSF) or filgrastim (non-glycosylated G-CSF) leads to a higher cell number of collected engraft able progenitor cells. Thus, we investigated harvesting efficiency of the licensed G-CSF preparations in mobilising peripheral stem cells in a randomised study. ⋯ Univariate variance analysis revealed that this effect was caused by male donors: more progenitors cells per kg BW of the donor (7.73 x 10(6) vs. 6.88 x 10(6); P < 0.017) and of the recipient (10.1 x 10(6) vs. 8.88 x 10(6), P < 0.029) could be harvested. There was no significant difference in the percentage of donors in whom a second aphaeresis was required (9.6% vs. 11.6%). Lenograstim mobilises progenitor cells into the peripheral blood more effectively in males than filgrastim.