The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease.

OBJECTIVE: To assess the long-term systemic and neurologic responses to enzyme replacement therapy (ERT) with macrophage-targeted glucocerebrosidase in patients with type 3 Gaucher's disease. STUDY DESIGN: Patients with type 3 Gaucher's disease (n = 21), aged 8 months to 35 years, were enrolled in a prospective study. Enzyme dose was adjusted to control systemic manifestations. Clinical and laboratory evaluations were performed at baseline and every 6 to 12 months thereafter. Patients were followed up for 2 to 8 years. RESULTS: Significant improvement in hemoglobin levels, platelet count, and acid phosphatase values occurred. Liver and spleen volume markedly decreased, and bone structure improved. Nineteen patients had asymptomatic interstitial lung disease unresponsive to ERT. Supranuclear gaze palsy remained stable in 19 patients, worsened in one patient, and improved in one. Cognitive function remained unchanged or improved over time in 13 patients but decreased in 8 patients, 3 of whom developed progressive myoclonic encephalopathy accompanied by cranial magnetic resonance imaging and electroencephalographic deterioration. CONCLUSIONS: At relatively high doses, ERT reverses almost all the systemic manifestations in patients with type 3 Gaucher's disease. Most treated patients do not deteriorate neurologically. Novel therapeutic strategies are required to reverse the pulmonary and neuronopathic aspects of the disease.

Policy and implementation of user fees in Jamaican public hospitals.

User fees and other forms of copayment for health care are becoming of increasing interest to policymakers in developing countries. As indigenous populations continue to expand in response to current and historically high fertility, and government resources become constrained due to macroeconomic circumstances, publicly provided health care is being squeezed financially. Most developing countries have committed their governments to either providing for all health care or at least ensuring that all citizens have access to health care regardless of ability to pay. This has translated in most contexts into blanket coverage for the entire population financed and generally provided by the government. Recent periods of slow growth, high debt burdens and restricted spending on high recurrent cost sectors, such as health care under International Monetary Fund and other donor agreements have reduced many developing countries' budgets and often the real value of health expenditures. The costs of inputs (personnel, drugs and consumables), however, have not declined and quality or quantity have been necessarily reduced. At the same time, options for financial relief outside the tax system have become of increasing interest to financially constrained governments. User charges are straightforward, easily understood and can in theory be profitable in the short term. From a fairness perspective, they also charge those who actually use the health system. Their major drawback for policymakers is the potential for undermining equity in the health system.