Acetazolamide for Monge's disease: efficiency and tolerance of 6-month treatment

Am J Respir Crit Care Med. 2008 Jun 15;177(12):1370-6. doi: 10.1164/rccm.200802-196OC. Epub 2008 Apr 3.


Rationale: 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.

Objectives: 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.

Methods: 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.

Measurements and main results: 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.

Conclusions: 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.

Trial registration: NCT00424970.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acetazolamide / adverse effects
  • Acetazolamide / pharmacology
  • Acetazolamide / therapeutic use*
  • Altitude Sickness / complications
  • Altitude Sickness / diagnostic imaging
  • Altitude Sickness / drug therapy*
  • Analysis of Variance
  • Carbonic Anhydrase Inhibitors / adverse effects
  • Carbonic Anhydrase Inhibitors / pharmacology
  • Carbonic Anhydrase Inhibitors / therapeutic use*
  • Cardiac Output / drug effects
  • Chronic Disease
  • Double-Blind Method
  • Echocardiography
  • Humans
  • Hypertension, Pulmonary / drug therapy
  • Hypoxia / drug therapy
  • Male
  • Middle Aged
  • Peru
  • Polycythemia / drug therapy*
  • Prospective Studies
  • Tricuspid Valve Insufficiency / etiology


  • Carbonic Anhydrase Inhibitors
  • Acetazolamide

Associated data