Along with the hematologic effects of sickle cell disease (SCD), numerous chronic organ injuries affect this patient population throughout the lifespan.1 Organ damage is associated with organ failure and premature death in individuals with SCD, underscoring the critical need for timely detection and treatment of these injuries.2
“Most organ injuries remain asymptomatic for several decades and can only be detected through early systematic screening,” researchers wrote in a review published in Haematologica.2 Optimizing efforts to prevent and manage these complications would likely improve quality of life in patients with SCD in addition to reducing the risk of early mortality. However, there is currently no established consensus regarding detection and prevention strategies for chronic organ damage in these patients.2
We interviewed the following experts to discuss recommended approaches for screening, prevention, and treatment of the various organ injuries affecting children with SCD: Derrick L. Goubeaux, DO, pediatric hematologist and oncologist and medical director of the Sickle Cell Program at Children’s Minnesota in Minneapolis; and Chris McKinney, MD, hematologist and oncologist at Children’s Hospital Colorado and assistant professor at University of Colorado School of Medicine in Aurora.
What are some of the common types of organ damage that affect pediatric patients with SCD?
Dr Goubeaux: SCD can impact any and all organs of the body, with this damage beginning in childhood. The spleen is one of the earliest organs to manifest its damage. Children with SCD are started on antibiotics for infection prophylaxis at just weeks of age due to the damage to the spleen by the sickle cells.3 This places these patients at higher risk for certain types of infections. Blood can also pool or sequester in the spleen, which can cause an acute decline in the hemoglobin traveling to other organs and cause further downstream damage and complications.
The normal blood flow through the major arteries of the brain can be impacted in a way that puts individuals with SCD at a significantly higher risk for neurological complications such as an acute stroke, as compared to the general population. The cardiopulmonary system can be impaired in how blood flows through the heart and blood vessels of the lungs. The blood vessels providing profusion to the retina can become damaged. These are just a few examples of the organ damage that can affect children with SCD.
Dr McKinney: The most common sites of chronic organ damage for patients with SCD include the brain, eyes, kidneys, spleen, heart, and bones. Overt stroke occurs in up to 11% of patients with hemoglobin SS disease and silent cerebral infarctions are found in 40% of patients by the age of 14 years.4,5 Sickle retinopathy can result from abnormal proliferation of small blood vessels in the back of the eye that are prone to hemorrhage, increasing the risk of retinal detachment or vision loss.
Chronic sickling within the small blood vessels of the kidneys can lead to a spectrum of progressively worsening renal disease ranging from urinary concentrating defects and proteinuria in children to hematuria, glomerular and renal tubular disease, and ultimately end-stage renal disease in adults. Most children with SCD have functional asplenia due to splenic autoinfarction, increasing their risk for sepsis or serious infections. Patients are also at increased risk of developing pulmonary hypertension and bone infarctions/osteonecrosis, ultimately leading to possible need for joint replacement.