Haitians have a high incidence of sickle cell disease (SCD), but limited healthcare resources. The Comparative Study of Children in Haiti and Miami with Sickle Cell Disease (CSHSCD) (NIH R01HL149121) assessed clinical and social characteristics of children of Haitian immigrants and African American controls and compared them with Haitian children living in Haiti. We hypothesized that there would be significant differences and that differences in the Haitian American group may be related to time since immigrating. For this report, baseline characteristics of a total of 255 children (mean age 38 months; 204 Haitians, 32 Haitian American and 19 African American) were compared as well as parental self-reported barriers to care and acculturation. Children with hemoglobin SS in Miami of either African American or Haitian ethnicities were more likely receiving hydroxyurea (p<0.001) and less likely have experienced a vaso-occlusive crisis (p<0.001) compared to children in Haiti. Being in the United States over 10 years was associated acculturation and better English proficiency. Children in Haiti were more anemic, more likely to be malnourished, and to have incomplete pneumococcal conjugate vaccinations. Haitian families faced issues affording treatment. Longer length of stay in the United States was associated with appropriate pneumococcal vaccination, higher parental education, and better English competency. Haitians living in Haiti were at a significant disadvantage in every aspect of care, requiring ongoing assistance. We encourage global partnerships to decrease barriers to care in disadvantaged populations.

Objectives: Transfusion-associated iron overload may cause liver fibrosis. We compared transient elastography (TE) and aspartate aminotransferase-platelet ratio index (APRI), non-invasive markers for hepatic fibrosis, to percutaneous liver biopsy in children and young adults with sickle cell disease (SCD). Methods: Subjects had serum ferritin was >500 ng/mL and lacked history of viral hepatitis. APRI scores, FerriScan® R2-MRI, liver biopsies, and TEs were obtained. Hepatic fibrosis was scored according to Metavir system. Age-matched controls with SCD but without iron overload were enrolled for APRI and TE assessments. Results: Nineteen subjects (cases: 12 males, 7 females), ages 10-21 years (mean 15.9 years), were transfused an average of 9.67 years, had a mean serum ferritin of 4,899±2849 ng/mL (range 693-11,604 ng/mL), and a liver iron concentration (LIC) of 8.46±3.95 mg/g dry liver weight by R2-MRI. Mean APRI was 0.33±0.13 (0.13-0.61) in cases and 0.27±0.10 in five controls. The mean liver stiffness measures (LSM) in cases, assessed by TE, was 8.46±3.95 kPa, ranging from 3.5-14.6 kPa (expected normal less than 7 kPa). Controls had a mean LSM of 5.72±1.74 kPa (4.6-8.7 kPa). Comparison of LSM to histological fibrosis for cases revealed a T-value of 6.94, p<0.0001. There was no significant correlation between APRI and histological fibrosis and between LSM and APRI or LSM and ferritin. Conclusion: TE measures but not APRI significantly correlated with histologic fibrosis. Although the small sample size limits generalization, LSM > 10 kPa suggests liver fibrosis in children and young adults with SCD and may merit histologic confirmation especially if persistent.

Sickle cell disease (SCD) requires coordinated, specialized medical care for optimal outcomes. There are no United States (US) guidelines that define a pediatric comprehensive SCD program. We report a modified Delphi consensus-seeking process to determine essential, optimal, and suggested elements of a comprehensive pediatric SCD center. Nineteen pediatric SCD specialists participated from the US. Consensus was predefined as 2/3 agreement on each element’s categorization. Twenty-six elements were considered essential (required for guideline-based SCD care), ten were optimal (recommended but not required), and five were suggested. This work lays the foundation for a formal recognition process of pediatric comprehensive SCD centers.