Clinical trial NCT05681598: Hydroxyurea Treatment for Adult Sickle Cell Anemia Patients in Kinshasa
Official title: Diagnosis and Treatment With Hydroxyurea of Sickle Cell Anemia in Democratic Republic of the Congo
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Sickle-Cell Anaemia
Keywords: sickle cell anemia
hydroxyurea
adult patients
kinshasa
Lead sponsor: University of Kinshasa
Sponsor class: OTHER
Collaborators: name: KU Leuven
First posted: 2023-01-12
Enrollment: 166
Sex: ALL
Minimum age (years): 18
Maximum age (years): 40
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: all eligible patients received during the study period were included and received treatment.
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: changes from baseline in body mass index (BMI) to year 2
timeFrame: baseline, year 1 and year 2
measure: changes from baseline in the frequency of Acute clinical events to year 2
timeFrame: baseline, year 1 and year 2
measure: changes from baseline in complete blood count to year 2
timeFrame: baseline, 3, 6, 9, 12, 15, 18, 21 and 24 months
measure: changes from baseline in Fetal hemoglobin to year 2
timeFrame: baseline, 3, 6, 9, 12, 15, 18, 21 and 24 months
measure: changes from baseline in left ventricle dilation to year 1
timeFrame: baseline, 4, 8 and 12 months
measure: changes from baseline in the thickness of left ventricular walls to year 1
timeFrame: baseline, 4, 8 and 12 months
measure: changes from baseline in the tricuspid regurgitation jet velocity to year 1
timeFrame: baseline, 4, 8 and 12 months
measure: changes from baseline in the pulmonary acceleration time (PaccT) to year 1
timeFrame: baseline, 4, 8 and 12 months
measure: Changes from baseline in E and A mitral to year 1
timeFrame: baseline, 4, 8 and 12 months
measure: Changes from baseline in E' mitral to year 1
timeFrame: baseline, 4, 8 and 12 months
measure: Changes from baseline in the E/E' mitral ratio to year 1
timeFrame: baseline, 4, 8 and 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Kinshasa
country: Congo, The Democratic Republic of the
facility: University of Kinshasa
Study officials:
affiliation: Center of human genetics. Faculty of medicine. University of Kinshasa
name: Prosper Lukusa Tshilobo
role: STUDY_DIRECTOR
References: