Small Study Group C Report from Benin Workshop

Hydroxyurea Therapy in Africa and other Developing Regions

Co-Chairs: Rajagopal Krishnamoorthy (France) and Richard Lottenberg (USA)

What plans are in place for the use of hydroxyura therapy in developing countries?

Previous and current major clinical trials of hydroxyurea (HU) therapy in SCD were discussed.

There is infrequent use of HU therapy in sub-Saharan Africa. However, certain medical centres in Africa have considerable interest in conducting formal studies e.g.

The clinical investigation of HU therapy in Africa would be of high interest to the countries represented. Even if cost issues can be effectively dealt with, an effort simply focused on implementation guidelines would not be appropriate at this time.

Given the absence, or limited availability, of disease modifying interventions, such as hematopoietic stem cell transplantation and chronic red cell transfusions, HU therapy really represents the viable option across practice settings in low- and mid-income countries and is worthy of appropriate clinical investigation.

What are the hurdles and approximate costs of introducing hydroxyurea therapy in developing countries?

Barriers to HU therapy were addressed according to the following categories:

What research is needed to introduce hydroxyurea therapy into the management of SCD in developing countries?

There are major concerns about using results from clinical trials in developed countries to guide therapy recommendations for less developed settings such as Africa. More areas of uncertainty include the impact of infections (e.g. malaria, tuberculosis) and differences in local environment (e.g. diet, lifestyle) on HU efficacy, risks and outcomes.

It is necessary to enroll patients in developing countries in clinical research. The question remains whether a placebo-controlled trial in Africa is necessary considering the results from the MSH trial in the USA and the challenges of conducting such a study.

The need for efficacy research (clinical trials in “ideal” settings) was differentiated from effectiveness research (studies in “real world“settings). There was enthusiasm for pursuing studies of effectiveness.

How can governments and funding agencies best be approached to provide support for the introduction of hydroxyurea therapy into developing countries?

SCD must be recognized and pursued as a public health priority in order to pursue adequate funding for SCD-related health care including infrastructure support for HU therapy.

Cost-effectiveness studies of HU therapy (possibly in comparison with chronic red cell transfusion) as well as studies demonstrating improvement in patient/family quality of life (particularly alleviation of pain) will strengthen the case for governments and funding agencies to support programs to implement HU therapy.