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Chronic organ injuries in SCD children
Apart from stroke prevention, the quality of evidence regarding the efficacy of actual screening strategies is still not high enough. Moreover, the ability of chronic transfu- sion, hydroxyurea and other drugs to prevent chronic organ failure has not been demonstrated by controlled studies. However, several encouraging studies do suggest a potential benefit of hydroxyurea to prevent degradation of renal function. The impact of new drugs such as crizan- luzimab and voxelotor on organ function is still under investigation. While there are still no individual predictive
Table 1. Screening tools in children and adolescents for the detection of chronic organ complications of sickle cell disease.
Organ
Heart
Pulmonary hypertension Myocardial ischemia
Lung and airways
Pulmonary disease Upper airway obstruction
Kidney
Renal failure
Brain
Cerebral vasculopathy Silent infarct
Liver
Cholangiopathy
Transfusion iron overload
Eye
Retinopathy
Hip
Osteonecrosis
of the femoral head
Screening tool
(apart from history and physical examination)
TRV on echocardiography ECG
Pulmonary function tests
ENT control
(+ polysomnography when necessary)
Blood pressure Creatinine, albuminuria
Transcranial doppler
Academic performance Neurocognitive screening Cerebral MRI
Liver tests
Abdominal ultrasonography
Ferritin
Liver iron content on hepatic MRI
Dilated fundoscopic examination
OCT
Radiography MRI
US recommendations for screening
In case of symptoms or suggestive signs
In case of symptoms or suggestive signs
In case of symptoms Yearly
Systematic screening
Yearly screening from age 2 to at least 16 years in SCA patients
No systematic MRI in asymptomatic children
No systematic screening
Systematic screening in chronically transfused patients
No consensus
In case of symptoms (hip pain)
Recommendations from a European panel of experts for screening
Yearly
Systematic in all children >5 years
In case of symptoms or suggestive signs Consider cardiac MRI
Yearly
Systematic in all children >5 years Yearly
Yearly systematic screening
Yearly screening from age 2 to at least 16 years in SCA patients
No consensus
Systematic MRI starting at 6 years old for some experts
Abdominal ultrasonography once a year from age 5
Systematic screening in chronically transfused patients
Yearly examination after 10 years old (6 years old for some experts)
Systematic radiography after
the age of 6 years, and then every 1-3 years according to the clinical findings
Evidence-based treatment (EBT)
No EBT
HU suggested
No EBT
HU suggested
Anti-asthmatic treatments
if necessary Adeno-/tonsillectomy if upper airway obstruction
No EBT
HU suggested if microalbuminuria ACE inhibitor suggested if proteinuria
Monthly transfusion in patients with velocities >200 cm/s
No EBT
HU suggested if silent infarct
No consensus
Elective cholecystectomy even in asymptomatic patients for some experts
Iron chelation
Laser photocoagulation if severe proliferative retinopathy
Conservative or operative treatment
factors, current strategy options may be to propose hydroxyurea to all HbSS and HbS/b0 children; this option has been considered by US and British experts.37 Another option may be to take the view that the potential severity of SCD in adults justifies proposing hematopoietic stem cell transplantation (and, in the future, gene therapy) even in asymptomatic patients. This debate is still ongoing, but it will be essential that patients help define their own health priorities and play a role in the design of clinical research.
ACE:angiotensin-converting enzyme;ENT:ear,nose and throat;HU:hydroxyurea;MRI:magnetic resonance imaging;OCT:optical coherence tomography;SCA:sickle cell anemia (patients with HbSS and HbS/b0-thalassemia genotypes); TRV: tricuspid regurgitant jet velocity.
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