Comprehensive Care Model: A Model for Sickle Cell Disease Management - PowerPoint Presentation

1. Comprehensive Care Model: A Model for Sickle Cell Disease Management Dr Doreen Karimi MutuaPediatric Hematologist/OncologistGertrude’s Children’s Hospital

2. OutlineHistoryStatus of Sickle Cell Disease Care Rationale of Comprehensive Care in Sickle Cell DiseaseLandmark studiesComponents of Comprehensive Sickle cell CareAction Plan for KenyaSummary

3. It’s 100 years already!!

4. From the Forefathers of Medicine: Hippocrates (460-377 B.C.E.) “Another sickness of the spleen. It comes on mainly in the springtime and is caused by the blood. The spleen becomes engorged with blood, which evacuates into the stomach. Shooting pains in the spleen, the breast, the clavicle, the shoulder, and beneath the shoulder blade. The body’s coloration resembles lead. Sores form on the leg and become large ulcerations. The discharges with the feces are bloody and bluish green. The belly hardens and the spleen is like a stone. This one is more murderous than the one before, and few survive it.”

5. Sickle Cell disease (SCD)SCD is the commonest haemoglobinopathy in the worldThere are about 300,000 children born with SCD every year, 70-75% are in AfricaEstimated life expectancy for SCD in high income countries is 45-50years but in low and Medium income countries more than 50 % of these children die before their 5th birthday70% of the deaths in SCD are preventableChallenges:  Lack of basic facilities to manage patients, absence of systemic screening for SCD, poor research, documentation, and intervention protocolsHsu L, Nnodu OE et al. White Paper: Pathways to Progress in Newborn Screening for Sickle Cell Disease in Sub-Saharan Africa. J Trop Dis Public Heath. 2018;6(2):260Wastnedge E, Waters D, Patel S.The global burden of sickle cell disease in children under five years of age: a systematic review and meta-analysis. J Glob Health. 2018 Dec;8(2):021103

6. Rationale of Comprehensive SCD careComprehensive care plan has led to a marked increase in survival for children with SCD It is now rare for children with SCD to die in developed countries, with a reported survival into adulthood of 94% in the United States and up to 99% in EnglandThe 59th World Health Assembly (WHA) in 2006, identified SCD as a significant public health problem in Africa that may contribute to up to 16% of under-5 mortality in some countriesWHA resolution 59.20 urged African nations with high burdens of SCD to design and implement national SCD programs with a focus onWidespread awareness-raisingEarly identification by Newborn screening(NBS)Early access to adequate preventative care, and training of medical professionals on SCD June 19th as World Sickle Cell Day: the United Nations issued a call for action among African nations to address the unmet burden of SCD

7. Land mark studies: Natural History of SCDCooperative Study on Sickle Cell Disease(CSSD)Natural history of SCD Newborn cohort- majority of deaths occurring in the first six months of lifeMajor events in children: infections, anemiaIncreased Hb F associated with mild disease 50% of adults died before 5th decade: acute pain, acute chest syndrome, strokeHydroxyurea use reduces pain episode by 45%, acute chest syndrome by 44% and blood transfusion by 30%

8. Land mark studies: Penicillin prophylaxisProphylatic Penicillin Study(PROPS)Despite universal coverage of the pneumococcal vaccine, Streptococcal infections significantly caused morbidity and mortality until Penicillin prophylaxis was introduced

9. Land mark studies: Hydroxyurea use in childrenPed- HUG trialBaby- HUG trial lower rates of initial and recurrent episodes of pain, dactylitis, acute chest syndrome, and hospitalization, Reduction in hemolysis, Increased Hb F,Reversible and predictable adverse effects of HydroxyureaBeneficial effects on splenic function preservation?effect on proteinuria, hypoxemiaNo safety concerns

10. Land mark studies: Stroke in Sickle Cell DiseaseSTOP TrialsSWiTCH trialTWITCHChronic transfusion in primary and secondary stroke preventionChronic transfusion and chelation therapy superior to Hydroxyurea/phlebotomy in stroke preventionHydroxyurea is effective in stroke prevention in patients with abnormal transcranial ultrasound velocity

11. Land mark studies: Hydroxyurea safe and effective for sickle cell anemia in African childrenREACH trial (Kenya amongst 4 countries involved) Among sub-Saharan African children with sickle cell anemia (SCA) treated with hydroxyurea, 5.1% experienced dose-limiting toxic events, well below the protocol-specified allowable rate of 30%. Compared to pretreatment rates, children treated with hydroxyurea had significantly reduced rates of vaso-occlusive pain, infection, malaria, transfusion, and death after one year.

12. Components of Comprehensive Care ModelComprehensive Care SCDNewborn screeningProphylactic Penicillin ProphylaxisHealth Education to FamiliesImmunization practicesNutritional supportMalaria infection preventionManagement of acute complicationsScreening for Chronic organ damageHydroxyurea usePsychosocial support

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14. Where do we begin?Kenya is listed amongst the countries with high burden for SCDIt has been shown that Newborn screening and link to comprehensive care centers is cost effectiveEarly introduction of penicillin prophylaxisHealth education to families during the patient visitsImmunization program already activeEnsure availability of Hydroxyurea Ensure availability of pain medication and safe blood productsEstablish multidisciplinary careEstablish guidelines on chronic organ damage screening: Urinalysis, Transcranial Doppler, Ophthalmology reviews, Iron overload screening, Echocardiography

15. SummaryChildren with SCD have the potential to live and let live with decreased morbidity and mortalityThis requires an adoption and commitment to comprehensive care approach to their managementLets hear a word from the Kenya Sickle Cell Foundation Chair!Thank You!