On-demand therapy enables stopping haemorrhages rapidly, reducing joint pain and restoring joint mobility, but does not prevent the beginning and subsequent development of haemophilic arthropathy. The main objective of this study was to identify the clinical and orthopaedic status of severe haemophilic patients with bleeding phenotype receiving on-demand treatment in Spain. We conducted an epidemiological, observational, retrospective study, recruiting 167 patients from 36 centres (92% of them with haemophilia A), median age at enrolment of 35 years. Forty per cent of the patients received a combination of on-demand and short-term prophylaxis regimen; the rest was under on-demand treatment. One hundred and forty-five patients (87%) reported at least one bleeding episode and 22 (13%) of the biologically severe patients had no bleeding phenotype. Seventy-one per cent of the studied population presented established haemophilic arthropathy, reaching 80% if we exclude patients without bleeding phenotype. Forty-three per cent of these patients had one or two joints affected, 28% of them had three or four affected joints, 20% reported five or six affected joints and 9% more than six injured joints. An increase in established haemophilic arthropathy with age was observed. Forty-six patients underwent orthopaedic surgery at least once. These data show that on-demand therapy is not effective in preventing the development of haemophilic arthropathy in severe haemophilic population with bleeding phenotype. Therefore, we suggest that the optimal treatment in these patients should be based on prophylaxis. We recommend analysing the reasons for ending prophylaxis, in case its reinstatement should be necessary.
The increase in oral anticoagulant (OA) treatment has led to development of new strategies for its control. We tested the efficacy of the GAO software (Dade Behring) in providing adequate acenocoumarol dosages for patients whose international normalised ratios (INRs) were no more than 0.5 points out of range. From December 1998 to August 1999, all outpatients in this setting were randomly assigned to receive computer-generated or traditionally fixed OA doses. Patients in their first 4 weeks of treatment were excluded. Overall, 8,352 dosages were calculated by the computer and 7,586 by the medical staff. The main endpoint of the study was the time spent in target INR range. The computer matched the traditional dosing, achieving a small but statistically significant greater efficacy in maintaining patients within the INR target range. The percentage of INR determinations over 5.5 was very low in both groups. Our results validate the computerised acenocoumarol dosing in our centre, achieving at least similar levels of effectiveness and safety compared with traditional dosage by medical staff.
Recently advances have been made in the treatment of acute leukemia in children, it is now possible to cure more than 70% of children with acute lymphoblastic leukemia. With the introduction of more intensive chemotherapy regimens in patients at higher risk of relapse and the identification of cases that could be less intensely treated to diminish long-term toxicity, it could be possible to improve these excellent results. In contrast, pediatric acute myeloid leukaemia seems to be a more heterogeneous disease and its response to conventional chemotherapy is not as uniform. Introduction of new and more efficacious therapies is necessary to improve the poor outcome, especially among patients with high-risk features.
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