ENERCA clinical recommendations for disease management and prevention of complications of sickle cell disease in children

Montalembert, Mariane de; Ferster, Alina; Colombatti, Raffaella; Rees, David C.; Gulbis, Béatrice

doi:10.1002/ajh.21865

Cited by 37 publications

(35 citation statements)

References 163 publications

Supporting

Mentioning

Contrasting

Unclassified

Order By: Relevance

“…The registry is a purely observational study that does not regulate patient treatment. However, national treatment guidelines recommend basic health care measures for SCD, such as parental education, vaccinations, the use of antibiotic prophylaxis or TCD screening as do international treatment guidelines . In Germany, everybody is covered under an obligatory health insurance system, regardless of their employment status.…”

Section: Methodsmentioning

confidence: 99%

Sickle cell disease in Germany: Results from a national registry

Kunz

Lobitz²,

Grosse³

et al. 2019

Pediatric Blood & Cancer

View full text Add to dashboard Cite

Background Limited data on the prevalence and medical care of sickle cell disease (SCD) in Germany are available. Here, we make use of a patient registry to characterize the burden of disease and the treatment modalities for patients with SCD in Germany. Procedure A nationwide German registry for patients with SCD documents basic data on diagnosis and patient history retrospectively at the time of registration. A prospective annual documentation provides more details on complications and treatment of SCD. For the current analyses, data of 439 patients were available. Results Most patients had homozygous SCD (HbSS 75.1%, HbS/β‐thalassemia 13.2%, and HbSC 11.3%). The median age at diagnosis was 1.9 years (interquartile range, 0.6‐4.4 years), most patients were diagnosed when characteristic symptoms occurred. Sepsis and stroke had affected 3.2% and 4.2% of patients, respectively. During the first year of observation, 48.3% of patients were admitted to a hospital and 10.1% required intensive care. Prophylactic penicillin was prescribed to 95.6% of patients with homozygous SCD or HbS/β thalassemia below the age of six and hydroxycarbamide to 90.4% of patients above the age of two years. At least one annual transcranial Doppler ultrasound was documented for 74.8% of patients between 2 and 18 years. Conclusion With an estimated number of at least 2000, the prevalence of SCD in Germany remains low. Prospectively, we expect that the quality of care for children with SCD will be further improved by an earlier diagnosis after the anticipated introduction of a newborn screening program for SCD.

show abstract

Section: Methodsmentioning

confidence: 99%

Sickle cell disease in Germany: Results from a national registry

Kunz

Lobitz²,

Grosse³

et al. 2019

Pediatric Blood & Cancer

View full text Add to dashboard Cite

show abstract

“…Hydroxyurea (HU) is widely recommended for treatment of children with SCD in developed countries . HU prolongs survival in children, as data from studies in Belgium and Brazil already reported .…”

Section: Introductionmentioning

confidence: 99%

“…HU prolongs survival in children, as data from studies in Belgium and Brazil already reported . It has a wide range of positive effects, from the reduction of painful vaso‐occlusive crisis (VOC) and acute chest syndromes (ACSs), the two most common acute manifestations of SCD, to the maintenance of stable velocities in the cerebral circulation . In spite of its established efficacy in childhood, population‐based data regarding its usage are scanty and information about its prescription and effectiveness in the real world come mainly from studies performed in adults or in the United States …”

Section: Introductionmentioning

confidence: 99%

Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

Colombatti

Palazzi²,

Masera

et al. 2017

Pediatric Blood & Cancer

Self Cite

View full text Add to dashboard Cite

show abstract

“…There is general agreement for using transfusion to prevent strokes in children, to treat severe acute chest syndrome, and as a preoperative precaution (de Montalembert, et al 2011, Habibi, et al 2015, Yawn, et al 2014). About 20% of SCD children in the US are on regular transfusion programmes; however, the use of transfusions in SCD is variable around the globe, as is the practice of chelation (Vichinsky, et al 2011a, Vichinsky, et al 2011b).…”

Section: Transfusion In Scdmentioning

confidence: 99%

How we manage iron overload in sickle cell patients

Coates

Wood

2017

Br J Haematol

View full text Add to dashboard Cite

Summary Blood transfusion plays a prominent role in the management of patients with sickle cell disease (SCD), but causes significant iron overload. As transfusions are used to treat the severe complications of SCD, it remains difficult to distinguish whether organ damage is a consequence of iron overload or is due to the complications treated by transfusion. Better management has resulted in increased survival, but prolonged exposure to iron puts SCD patients at greater risk for iron-related complications that should be treated. The success of chelation therapy is dominated by patient adherence to prescribed treatment; thus, adjustment of drug regimens to increase adherence to treatment is critical. This review will discuss the current biology of iron homeostasis in patients with SCD and how this informs our clinical approach to treatment. We will present the clinical approach to treatment of iron overload at our centre using serial assessment of organ iron by magnetic resonance imaging.

show abstract

ENERCA clinical recommendations for disease management and prevention of complications of sickle cell disease in children

Cited by 37 publications

References 163 publications

Sickle cell disease in Germany: Results from a national registry

Sickle cell disease in Germany: Results from a national registry

Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

How we manage iron overload in sickle cell patients

Contact Info

Product

Resources

About