Journal Article

Acetazolamide for Monge's Disease: Efficiency and Tolerance of 6-Month Treatment

Pham, Isabelle, Macarlupu, Jose-Luis, Privat, Catherine, Maignan, Maxime, Petitjean, Olivier, Richalet, Jean-Paul, Rivera-Ch, Maria, Leon-Velarde, Fabiola

2008

Overview

Monge's disease is characterized by an excessive erythrocytosis, frequently associated with pulmonary hypertension, in high-altitude dwellers. It has a considerable impact on public health in high-altitude regions. A preliminary study demonstrated the efficiency of acetazolamide (Acz) (250 mg/d for 3 wk) in reducing serum erythropoietin and hematocrit. Evaluate the efficacy and tolerance of a 6-month treatment with 250 mg Acz that could be chronically implemented and its effects on pulmonary artery pressure and cardiac function. A two-phase study was performed in patients (hematocrit > or = 63%) from Cerro de Pasco, Peru (4,300 m). First phase: a double-blind, placebo-controlled study in 55 patients who received a single dose of either 250 mg Acz (n = 40) or placebo (n = 15) by daily oral administration for 12 weeks. Second phase (open label): after a 4-week washout period, all patients received 250 mg Acz for 12 weeks. Hematocrit, blood gases, clinical outcome, and pulmonary artery circulation were evaluated. First phase: Acz decreased by 44% the number of polycythemic subjects (P = 0.02), decreased hematocrit from 69 to 64% (P < 0.001), and increased arterial O(2) pressure from 42 to 45 mm Hg (P < 0.001). No severe adverse effect or hypokalemia was recorded. The second phase reproduced the effects observed during the first phase, without cumulative effects on hematocrit. A 4-week washout restored basal hematocrit. Only patients who received Acz for 6 months showed a clear reduction in pulmonary vascular resistance. Acz reduces erythrocytosis and improves pulmonary circulation in Monge's disease without adverse effects. Its implementation as a chronic treatment for this disease appears efficient and safe.

• Publisher: Am Thoracic Soc,American Lung Association,Oxford University Press
• Subject: Acetazolamide - adverse effects / Acetazolamide - pharmacology / Acetazolamide - therapeutic use / Altitude / Altitude Sickness - complications / Altitude Sickness - diagnostic imaging / Altitude Sickness - drug therapy / Analysis of Variance / Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy / Biological and medical sciences / Carbonic Anhydrase Inhibitors - adverse effects / Carbonic Anhydrase Inhibitors - pharmacology / Carbonic Anhydrase Inhibitors - therapeutic use / Cardiac function / Cardiac Output - drug effects / Chronic Disease / Creatinine / Degenerative and inherited degenerative diseases of the nervous system. Leukodystrophies. Prion diseases / Disease / Double-Blind Method / Echocardiography / Headaches / Humans / Hypertension, Pulmonary - drug therapy / Hypoxia / Hypoxia - drug therapy / Intensive care medicine / Male / Medical sciences / Middle Aged / Neurology / Peru / Plasma / Polycythemia - drug therapy / Potassium / Prospective Studies / Public health / Pulmonary arteries / Pulmonary hypertension / Tricuspid Valve Insufficiency - etiology / Velocity