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Sickle cell anemia in sub-Saharan Africa: advancing the clinical paradigm through partnerships and research

Patrick T. McGann, Arielle G. Hernandez and Russell E. Ware

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Tables

  • Table 1.

    Sickle cell guidelines and recommendations with relevance for sub-Saharan Africa

    TopicGoalAge rangeApplicable to AfricaFeasible for AfricaNeed for more testingCurrent trials
    ScreeningDiagnosis
     Electrophoresis by IEF, HPLC, CZENeonates, teens, young adultsYesYesNoNo
     Point-of-care testingNeonates, teens, young adultsYesYesYesYes
    EducationEarly recognition
     Splenic sequestration<2 yYesYesNoNo
     Fever managementAll agesYesYesNoNo
    Preventive careProphylaxis
     AntimalarialsAll agesYesYesYesYes
     Insecticide-treated bed netsAll AgesYesYesNoNo
     Penicillin<5 yYesYesNoNo
     Vaccination<5 yYesYesNoNo
     Antibiotics for feverAll agesYesYesNoNo
     Blood culture with feverAll agesYesNoNoNo
    ScreeningEarly identification
     TCD ultrasound2-16 yYesYesYesYes
     ProteinuriaAll agesYesYesYesYes
     Cardiac function (TR Jet velocity)AdultsPossiblyPossiblyYesNo
    TreatmentTherapeutic
     TransfusionsAll agesYesYesYesNo
     HydroxyureaAll agesYesYesYesYes
     TransplantationAll agesNoNoNoNo
  • Table 2.

    Therapeutic options for SCA with potential relevance for sub-Saharan Africa

    TreatmentAdvantages and indicationsDisadvantages and challenges
    Erythrocyte transfusions• Treatment of severe anemia due to splenic sequestration, parvovirus infection, or malaria• Lack of sufficient blood donors
    • Additional oxygen-carrying capacity for life-threatening acute vaso-occlusion and organ damage• Infection transmission (HIV, hepatitis B and C, syphilis)
    • Effective treatment option for stroke and other neurologic complications• Erythrocyte alloimmunization
    • Inability to prepare blood components
    • Eventual need for iron chelation
    Hydroxyurea• Reduction of acute vaso-occlusive complications (pain, acute chest syndrome)• Limited drug availability
    • Oral administration• High cost relative to daily wages
    • Once-daily dosing• Optimal dosing not yet determined
    • Documented laboratory and clinical efficacy and efficacy• Cost and feasibility of routine laboratory monitoring, including WBC differential and reticulocytes
    • Low cost compared with alternatives• Inability to measure quantitative %HbF
    Stem cell transplantation• Potential cure• Lack of facilities and clinical expertise
    • Availability of full siblings, which increases the chance of HLA matching
    • Limited technology for HLA typing, cell processing, and preparation
    • Inadequate supportive care (antibiotics, transfusions, isolation rooms)
    • High risk of morbidity (graft versus host disease) and mortality
    • Extremely high cost
    • HLA, human leukocyte antigen; WBC, white blood cell.

  • Table 3.

    Current prospective research trials using hydroxyurea therapy for SCA in sub-Saharan Africa

    Clinical trialClinicalTrials.gov and statusPerformance sitesNo. of participantsStudy end points
    Sickle Cell Disease–Stroke Prevention in Nigeria Trial (SPIN)NCT01801423Kano, Nigeria40TCD velocity
    Active, not recruiting
    Realizing Effectiveness of Hydroxyurea Across Continents (REACH)NCT01966731Luanda, Angola; Kinshasa, DRC; Kilifi, Kenya; Mbale Uganda600Adherence, toxicity, dosing
    Recruiting
    Novel use Of Hydroxyurea in an African Region with Malaria (NOHARM)NCT01976416Kampala, Uganda200Malaria events
    Active, not recruiting
    Risk Clinical Stratification of Sickle Cell Disease in Nigeria: Assessment of Efficacy/Safety of Hydroxyurea TreatmentNCT02149537Ibadan, Nigeria40Cytopenia
    Enrolling by invitation
    Primary Prevention of Stroke in Children With SCD in Sub-Saharan Africa II (SPRING)NCT02560935Kano, Nigeria440Stroke occurrence
    Recruiting
    Low Dose Hydroxyurea for Secondary Stroke Prevention in Children With Sickle Cell Disease in Sub-Saharan Africa (SPRINT)NCT02675790Kano, Nigeria60Stroke recurrence
    Not yet recruiting
  • Table 4.

    Recommended approaches to partnerships and research opportunities in sub-Saharan Africa

    1. Involve local leaders as collaborators, especially from academia and government.
    2. Create partnerships that emphasize training and local capacity building.
    3. Conduct prospective research to ensure results are evidence-based.
    4. Follow rigorous ethical and research standards, to protect the rights of potentially vulnerable study participants.
    5. Train and use local personnel whenever possible, increasing in-country expertise and limiting exportation of samples and data.