Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa

University of Kinshasa

Status: Completed

Conditions: Sickle-Cell Anaemia

Treatments: Drug: Hydroxyurea

Study type: Interventional

Funder types: Other

Identifiers

NCT05681598

ZRDC2015PR088

Details and patient eligibility

About

The goal of this clinical trial is to evaluate the efficacy of hydroxyurea (HU) in improving disease severity in adult patients with sickle cell anemia in Kinshasa (Democratic Republic of Congo). This study aims to:

• assess the safety and efficacy of HU treatment in the Congolese environment;
• assess the reversibility of chronic cardiac lesions. Participants will take hydroxyurea for two years. The effects of the treatment will be evaluated periodically by clinical evaluation, biological tests, and echocardiographic exploration.

Full description

Sickle cell disease is common in sub-Saharan Africa, particularly in the Democratic Republic of the Congo (DRC). It is characterized by chronic hemolytic anemia with the need for transfusions, painful osteoarticular crises, and chronic organ damage, including the heart.

Hydroxyurea (HU) is a drug widely used in sickle cell patients in wealthy countries, while it is little used in poor countries with a high incidence of the disease.

This prospective study will focus on homozygous sickle cell participants, naïve to HU treatment. Participants will take HU in gradually increasing doses. They will be monitored for the possible occurrence of side effects. The effectiveness of the treatment will be evaluated according to the reduction in painful crises, and the need for blood transfusion; as well as based on biological changes and the reversibility or stabilization of cardiovascular complications.

Enrollment: 166 patients

Sex: All

Ages: 18 to 40 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• sickle cell anemia confirmed by DNA testing;
• moderate to severe clinical severity of sickle cell anemia;
• fetal hemoglobin lower than 15%.

Exclusion criteria

• poor compliance to follow-up consultation during the observational year
• participant already treated with HU
• pregnant women
• breastfeeding women
• congenital heart disease
• pulmonary disease

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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166 participants in 1 patient group

Hydroxyurea treatment

Experimental group

Description:

participants were treated with hydroxyurea

Treatment:

Drug: Hydroxyurea