Texas Children's Global Health New Initiatives

A New Day for Children with sickle cell disease

A New Day for Children with Sickle Cell Disease is an initiative of Texas Children’s Global HOPE to roll out large-scale sickle cell disease care for children in Africa by modeling integration of essential high impact interventions, such as infant screening, immunizations, penicillin prophylaxis, and hydroxyurea therapy) for sickle cell disease into primary child health services. The initiative is the brainchild of the experience and vision of pediatric hematologists we have trained in Africa and the experience of Baylor College of Medicine Children’s Foundations in African child and maternal health services.

What is sickle cell disease?

Sickle cell disease is an inherited genetic disease that causes red blood cells to deform, crumble, and clog blood vessels.

Sickle cell disease is a life-long condition that can cause severe damage to all organs in the body resulting in unbearable distress, disability, and death. Sickle cell disease, therefore, also has far-reaching consequences on the development and mental health of affected children and the wellbeing of their families.  

Why does sickle cell disease predominantly affect people of African ancestry?

• About 85% of people living with sickle cell disease are of African ancestry – whether living in Africa or in the diaspora.
• Every year, about 340,000 children in sub-Saharan Africa are born with sickle cell disease.
• It is estimated that about 15 million people are living with sickle cell disease in sub-Saharan Africa.
• The sickle cell gene abnormality is very common among Africans compared to other peoples around the world because individuals with the sickle cell gene but not the disease, i.e., carriers, are resistant to severe malaria. Over millennia, the gene abnormality became increasingly common in Africa because carriers were surviving the deadly effects of malaria. 
• Between 10–40% of people of central African ancestry have the sickle cell gene. This is the group that would benefit from the gene’s protective effects against malaria. 
• 1–2% of people of central African ancestry have two sickle cell genes, a situation that results sickle cell disease – a serious, potentially fatal condition.  

Which countries are most affected by sickle cell disease?

Nigeria, Democratic Republic of Congo, Tanzania, Uganda, and Angola are the five countries that have the highest burden of sickle cell disease in the world.

^{Adapted from Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573}

What interventions are effective and proven in reducing the complications of sickle cell disease?

The death rate of children born with sickle cell disease in the United States has dramatically reduced since the 1980s. Similar outcomes have been reproduced in other high-income countries and in small scale pilot projects or research studies using the following interventions: 

• Early detection of infants with sickle cell disease by routine universal infant screening, 
• Prevention of severe childhood infections by diligent routine childhood immunizations,
• Prevention of blood stream bacterial infections, particularly from the pneumococcus bacteria, by taking a daily of penicillin by mouth until the child is five years old, and,
• Taking a daily pill of hydroxyurea – a drug that reduces the amount sickle cells in blood to mitigate most of the complications of the disease.

Why haven’t African countries widely implemented interventions to control sickle cell disease?

• Despite the proven effectiveness of technically simple interventions, (i.e., infant screening, immunizations, penicillin prophylaxis, and disease modification with hydroxyurea) most children with sickle cell disease in sub-Saharan Africa die by their 5th birthday due to uncontrolled complications of the disease. 
   
• Sickle cell disease contributes about 16% to under-5 mortality in sub-Saharan Africa. In high income countries like the United States, over 95% of children with sickle cell disease grow to adulthood. Their average lifespan is 53–55 years. 
   
• Many research trials and pilot projects have demonstrated that proven interventions for sickle cell disease control are equally effective and safe in sub-Saharan Africa as in the United States.
   
• Despite these experiences, sickle cell disease care in sub-Saharan Africa remains tragically limited. A tiny minority of children—typically with devastating, irreversible complications—receive care in hospitals. Most affected children are never diagnosed.
   
• Systematic, large-scale delivery of proven public health interventions to control sickle cell disease care in sub-Saharan Africa is urgently needed but has never materialized because dedicated resources for sickle cell disease programs are scarce amidst competing health priorities.

What is Texas Childrens' strategy to catalyze transforming the outcomes of children with sickle cell disease in sub-Saharan Africa?

Guided by the experience and vision of our African partners and pediatric hematology experts we trained, Texas Childrens' strategy for rapid scale- up of systematic sickle cell disease care in sub-Saharan Africa is to integrate the essential interventions (i.e., infant screening, immunizations, penicillin prophylaxis, and hydroxyurea therapy) in primary health services, particularly using the health services expertise, systems, and infrastructure that have effectively controlled pediatric HIV in these countries.

We recognize the partnerships, experience, and infrastructure built by African governments, many global development agencies, and academic medical centers—such as Texas Children’s/Baylor College of Medicine—over the last several decades as a golden opportunity that should be leveraged to reduce the burden of sickle cell disease—rapidly and cost-effectively.

Our approach departs from the status quo of slow and costly attempts to establish vertical sickle cell disease delivery systems and infrastructure, which have not materialized.

Rather than building a separate system from the ground up, we are integrating sickle cell disease-specific skill sets, medical supplies, and custom digital innovations into the existing workforce, facilities, workflows, supply chains, and grassroots engagement structures.

What are we doing to implement Texas Children’s strategy for rapid scale up of systematic sickle cell disease care in sub-Saharan Africa?

Galvanizing global and pan-African partnerships

We have assembled a synergistic coalition of African and global health agencies, pharmaceutical companies, philanthropic foundations, and sickle cell community-based associations to lead the implementation of this strategy.  

Training the leaders of sickle cell disease care in Africa

Over the last seven years, we have trained 35 African pediatric hematologists in formal university-based, accredited clinical fellowship programs. These specialists are the pioneers of the field in Africa. They are guiding sickle cell disease policy for their countries’ Ministries of Health, conducting research, and designing training programs for primary health workers. Most importantly, they are megaphones for children and families impacted by sickle cell disease.

Integrating sickle cell disease care in primary maternal/child health services by leveraging pediatric HIV care models

Proven interventions to control sickle cell disease parallel those for pediatric HIV infection, namely (1) early infant screening/diagnosis, (2) prevention and treatment of opportunistic infections, and (3) suppression of HIV with anti-retroviral therapy. Also, pediatric HIV approaches are a prototype for delivery of sickle cell disease care because they are first experience of African health systems systematically delivering lifelong care for children with complex diseases on a large scale. 

The Baylor College of Medicine Children’s Foundations are our affiliated but locally led organizations in Africa that spearheaded systematic pediatric HIV services on the continent. Together as a network, these Foundations are the single largest provider of pediatric HIV care in the world. We are leveraging these sister institutions to apply their experience and infrastructure in rolling out the key components of sickle cell disease care:

• Universal screening of infants
  The AfroSickleNet White Paper on sickle cell disease screening in sub-Saharan Africa recommends point-of-care tests as the primary mode of testing because centralized laboratory-based screening programs have so far proven to be unaffordable and logistically not feasible in the African health systems context. There are now two trialed and proven point-of-care screening kits for sickle cell disease on the market. Pediatric HIV models across Africa have established systems for screening infants at birth and routine immunization visits. These screening services have 5Cs of quality: Confidentiality, Consent, Counseling, Correct test results, and Connections to appropriate treatment services for those that have positive screening results.
   
• Treatment and psychosocial care in primary care settings
  There are differences between countries in the capabilities of various health facilities levels. Overall, the bulk of pediatric HIV treatment in Africa occurs in primary health care facilities, with supervision support from more centralized secondary and tertiary facilities. Primary health care facilities are typically capable of providing (1) routine childhood immunizations, (2) prescribing and monitoring side effects of routine oral medications, (3) clinical assessment of children including growth & development monitoring, and referral in complicated situations, and (4) engaging families and communities to ensure retention in care, adherence to care plans, psychosocial and dignity support.
   
• Program monitoring and evaluation 
  Primary health care facilities are supported to collect, store, share, and respond to data for program management and improving quality of care.
   
• Education
  We offer an online course for healthcare workers involved in the care of children in Africa to develop competency in delivering evidence-based, essential sickle cell disease care. 
   

• Innovating digital solutions to coordinate sickle cell disease services
  Robust information management and communication systems underlie all aspects of public health services including screening programs. Through a US National Institutes of Health funded research grant and in partnership with Makerere University in Uganda, we have developed a cell phone digital app and digital system to facilitate coordination and monitoring & evaluation of sickle cell disease services in the African setting. 
  
  The rationale for creating the Sickle Cell Disease Information Management and Communication System (SIMCS) was the realization that whereas point-of-care tests are advantageous for large-scale screening in Africa, they require error-proof capture and sharing of information within and between the multitude of screening and treating health facilities, and the Ministry of Health central coordination and logistics offices. The SIMCS automates this process.
  
  The SIMCS is built on Uganda’s Ministry of Health extant digital infrastructure to ensure optimal crosstalk and sustainability. The app was assembled using off-the-shelf software that is secure and compatible with iOS and Android mobile phone operating systems. It uses Artificial Intelligence (AI) to help health workers interpret point-of-care tests; it has 99.4% accuracy in interpreting the tests.