Mortality and causes of death in children with sickle cell disease in the Netherlands, before the introduction of neonatal screening

Abstract

Summary This study analyzed the mortality and causes of death in sickle cell disease patients in the Netherlands, to provide a baseline for monitoring the effect of the recently introduced neonatal screening programme and to indicate areas of improvement in the care for these patients. All children (<18 years) diagnosed with sickle cell disease in a tertiary hospital from 1985 to 2007 were included. Vital status was determined up to March 2008. A total of 298 children were included: 189 (63%) patients had HbSS, 17 (6%) HbSβ0 thalassaemia, 72 (24%) HbSC and 20 (7%) HbSβ+ thalassaemia. Twelve patients (4%) died during a total follow‐up of 3896 patient years. All known deaths were sickle cell disease‐related. Meningitis/sepsis (n = 4; 33%), stroke (n = 3; 25%) and death during a visit to the country of origin (n = 3; 25%) were the most common causes of death. The overall mortality rate was 0·27 deaths/100 patient years [95% confidence interval (CI): 0·15–0·43]. The estimated survival at the age of 18 years was 97·3% (95% CI: 95–99%). This report confirms that the burden of mortality in sickle cell disease is increasingly shifting to adults. It is recommended that compliance to antibiotic prophylaxis, thorough counselling and support for patients travelling abroad and specialized peri‐operative care should receive continuous attention.

Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel

“…[58][59][60][61][62][63] Survival has improved significantly in the last two decades and 94% of children with SCD now survive until the age of 18 years thanks to better surveillance, pneumoccocus vaccination, penicillin prophylaxis and treatment with hydroyurea. 64,65 However, mortality is still significant once patients reach adulthood.…”

“…64,65 However, mortality is still significant once patients reach adulthood. 61,66 SCD-associated morbidity and mortality in young adults is largely due to as yet unpreventable complications such as priapism, avascular necrosis, chronic pulmonary impairment, hypertension, stroke and recurrent venoocclusive crises. 58,[67][68][69][70] The only curative approach for SCD is HSCT.…”

Lessons Learned From Talking With Parents About the Role of Hematopoietic Stem Cell Transplantation in the Treatment of Children With Sickle Cell Disease

“…[1][2][3]12,13 Whether parents in developed countries now perceive the risks of SCD in terms of acute life-threatening events in childhood, decreased life expectancy in adulthood, or decreased overall quality of life is not known. In our study, 65% of parents knew an adult with SCD and 66% reported that seeing adults with SCD made them worry about the quality of their child's future health.…”

“…The resulting deformed red blood cells cause anemia and chronic endothelial damage, leading to a variety of acute medical complications (e.g., acute chest syndrome, splenic dysfuction, and stroke), as well as chronic issues (e.g., pain, renal insufficiency, and bony destruction). [1][2][3][4] SCD is a global public health problem, afflicting millions worldwide and more than 80 000 people in the United States alone. [4][5][6][7] Without proper education and prophylactic care, SCD can be life-threatening, particularly early in childhood.…”

“…[6][7][8][9] However, advances in care have reduced childhood mortality from SCD in developed countries dramatically, with survival at 18 years now reaching 97%. 1,3 Morbidity and mortality in adulthood, however, remain high, particularly at the transition from pediatric to adult care. 3,10 The natural history of SCD continues to be characterized by chronic anemia, recurrent pain episodes, increased risk of life-threatening events such as stroke, cumulative organ damage, and reduced life expectancy.…”