Treatment of High Altitude Polycythemia by Acetazolamide

Condition(s) targeted: High Altitude Polycythemia

Intervention: acetazolamide (Drug)

Phase: Phase 4

Status: Not yet recruiting

Sponsored by: Association pour la Recherche en Physiologie de l'Environnement

Official(s) and/or principal investigator(s):
Jean-Paul Richalet, MD, PHD, Principal Investigator, Affiliation: ARPE, University Paris 13
Fabiola Leon-Velarde, PHD, Study Director, Affiliation: University Cayetano Heredia, Lima, Peru

Overall contact:
Maria Riveira, Email: mrivera@upch.edu.pe

The prevalence of High Altitude Polycythemia (or Chronic Mountain Sickness) is between 8 and 15% in the high altitude regions of South America. There is no pharmacological treatment available. After a first preliminary study in 2003 demonstrating the beneficial effects of acetazolamide in reducing hematocrit in these patients, after 3 weeks of treatment, we want to confirm this effect and implement a treatment protocol of 3 month-duration.

Official title: Hypoventilation and High Altitude Chronic Polycythemia: Acetazolamide as a Possible Treatment

Study design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Primary outcome:

Hemoglobin concentration after 3 months of treatment

Hematocrit after 3 months of treatment

Secondary outcome:

Systolic pulmonary arterial pressure after 3 months of treatment

Pulmonary vascular resistance after 3 months of treatment

Arterial oxygen saturation at rest after 3 months of treatment

Clinical score of Chronic Mountain Sickness after 3 months of treatment

Quality of lofe score after 3 months of treatment

Detailed description: Chronic mountain sickness (CMS) is characterized by an excessive number of red cells in the blood of persons living permanently above the altitude of 2,500m. The symptoms of this very incapacitating disease are : headaches, chronic asthenia, digestive troubles, sleep disturbances. The hemoglobin concentration is higher than 21 g/dl of blood. In addition, patients show a pulmonary hypertension of variable degree, as well as a systemic hypertension.

This disease affects essentially males, but women are also concerned after menopause. The evolution of the disease is always very dramatic, towards a cardiac failure and cerebral vascular stroke. The prevalence is between 8% and 15% on the Andean Altiplano . No pharmacological treatment is available.

A preliminary study was performed (Richalet et al. AJRCCM, 2005) that demonstrated the efficiency of acetazolamide (a carbonic anhydrase inhibitor) in reducing the hematocrit and the erythropoetin concentration,and increasing nocturnal oxygen saturation in patients suffering from CMS, after 3 weeks of treatment.

We plan to perform a double-blinded placebo-controlled study to evaluate the efficiency of a 3-month treatment with daily 250 mg acetazolamide to reduce the hematocrit and hemoglobin concentrations and ameliorate the clinical symptoms of 55 patients suffering from CMS and living at high altitude (Cerro de Pasco, Peru).

Minimum age: 18 Years. Maximum age: 65 Years. Gender(s): Male.

Criteria:

Inclusion Criteria:

- patients with Chronic mountain sickness and Hb > 21g/dl

Exclusion Criteria:

- patients smokers

- patients with respiratory or cardiovascular or renal disease

Maria Riveira, Email: mrivera@upch.edu.pe

University Cayetano Heredia, Lima 100, Peru

Related publications:

Richalet JP, Rivera M, Bouchet P, Chirinos E, Onnen I, Petitjean O, Bienvenu A, Lasne F, Moutereau S, Leon-Velarde F. Acetazolamide: a treatment for chronic mountain sickness. Am J Respir Crit Care Med. 2005 Dec 1;172(11):1427-33. Epub 2005 Aug 26.

Starting date: January 2007
Ending date: February 2007
Last updated: January 19, 2007