Primary Prophylaxis in Sickle Cell Disease: Is It Feasible? Is It Effective?

Thompson, Alexis A.

doi:10.1182/asheducation-2011.1.434

Cited by 6 publications

(4 citation statements)

References 65 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Since the first report of the disease by (Herrick, ), volumes have been written describing the clinical course and complications of the disease. It is well established that some of these, such as renal dysfunction and neuropsychiatric abnormalities, begin in infancy, with others, such as avascular necrosis, chronic lung disease and pulmonary hypertension, manifesting by the second and third decades of life (Thompson, ). Children have chronic anaemia, impaired growth, episodic pain crises, and many suffer life‐threatening complications, such as splenic sequestration, ACS and infarctive strokes.…”

Section: Progressive Course and Complicationsmentioning

confidence: 99%

Sickle cell disease: time for a closer look at treatment options?

2013

View full text Add to dashboard Cite

SummaryTremendous progress has been made in the care of individuals with sickle cell over the past several decades. Major successes have been comprehensive infection prophylaxis, prediction and prevention of stroke, and better transfusion care, the latter including both prevention of alloimmunization and treatment of iron overload. However, definitive therapies remain limited to hydroxycarbamide (hydroxyurea) and stem cell transplantation, both of which have been in use for at least two decades. Despite knowing the progressive natural history of the disease with organ dysfunction, failure, and ultimately death at a young age, definitive therapies are considered for only a small proportion of individuals. Consequently, while life expectancy has improved dramatically from the last century, the ongoing pace of advancement has slowed or stalled. We believe that it is time to broaden the use of definitive therapy for those with asymptomatic disease, being cautiously more aggressive in our approach.

show abstract

Section: Progressive Course and Complicationsmentioning

confidence: 99%

Sickle cell disease: time for a closer look at treatment options?

2013

View full text Add to dashboard Cite

show abstract

“…In sickle cell patients low stable plasma haemoglobin is more likely in patients who have restricted lung function, who are older and who have more systemic inflammation and low BMI. Recently molecular markers rather than biophysical markers of severity have received most attention in sickle cell disease [28]. Throughout the last century, sickle cell disease has been studied at several physiologic levels (molecular, tissue, cellular and biochemical) little consensus has emerged on markers of disease severity [29,30].…”

Section: This Is the First Study Of Lung Function And Inflammation In Sicklementioning

confidence: 99%

Lung Function and Inflammation in Adult Sickle Cell Disease in a West Indian Sickle Cell Cohort

Williams¹

2012

J Pulmon Resp Med

View full text Add to dashboard Cite

Background:The commonest cause of mortality in sickle cell disease (SCD) is an acute condition called the acute chest syndrome. Little is known of the long term effects of this condition or of its effect on lung function.Objective: To determine lung function in sickle cell disease (SCD) adults and its relation to acute chest syndrome, baseline haemoglobin and systemic inflammation as estimated by serum C-reactive protein. Methods:The lung function in sickle cell disease (SCD) outpatients was compared to that in asymptomatic age and sex matched controls. Stable haemoglobin and serum C-reactive protein (CRP) were measured.Results: 74 controls of mean age 31 years and 154 sickle cell disease (SCD) outpatients with mean age 31 with 44% males were recruited. Controls and sickle cell disease (SCD) patients did not differ in age or sex. 18% sickle cell disease (SCD) patients had at least one episode of acute chest syndrome and 7% had two or more episodes. Forced vital capacity was lower in sickle cell disease (SCD) patients than controls and 47% of sickle cell disease (SCD) patients had a restrictive ventilatory defect. A further 19% had obstruction but the presence of this abnormality was not related to smoking habit. Patients with a history of two or more acute chest syndrome episodes were more likely to have poor lung function. Haemoglobin was positively correlated with forced vital capacity and inversely correlated C-reactive protein. Multivariate analysis showed that haemoglobin in sickle cell disease (SCD) patients was lower if patients also had a low forced vital capacity, high serum C-reactive protein, low body mass index, older age or male gender.Conclusions: Abnormal lung function is common in sickle cell disease (SCD) adults. In sickle cell disease (SCD), adult's low haemoglobin is related to a restrictive ventilatory defect and systemic inflammation. Severity of stable disease in sickle cell patients is related to poor lung function and systemic inflammation.

show abstract

“…The assessment of the GFR is key to identifying opportunities to prevent or provide early interventions to patients with SCD ( 10 ) . Knowledge of factors that impose higher risk is an important way to guide prevention and early intervention.…”

Section: Discussionmentioning

confidence: 99%

Glomerular filtration rate is altered in children with sickle cell disease: a comparison between Hb SS and Hb SC

Pereira¹,

Nascimento²,

Sousa³

et al. 2013

Revista Brasileira De Hematologia E Hemoterapia

View full text Add to dashboard Cite

BackgroundRenal failure is common among older patients with sickle cell disease; this is preceded by subclinical glomerular hyperfiltration. Data about renal function of adults with sickle cell disease have been reported, but data on children is scarce, especially when comparing heterozygotic and homozygotic patients. ObjectiveThe goal of this study was to investigate the glomerular filtration rate of heterozygotic and homozygotic children with sickle cell disease. MethodsThe glomerular filtration rate of 11 children with sickle cell disease [7 homozygotic (SS) and 4 heterozygotic (SC)] with a mean age of 11 years (standard deviation: ± 5 years) was evaluated using standard laboratory techniques. Results are presented as descriptive analysis. ResultsOur results suggest that glomerular hyperfiltration is present in children with sickle cell disease; this is more evident in homozygotic than heterozygotic children. ConclusionThere is evidence of a need to monitor the renal function of children with sickle cell disease when special attention should be paid to homozygotic patients.

show abstract

Primary Prophylaxis in Sickle Cell Disease: Is It Feasible? Is It Effective?

Cited by 6 publications

References 65 publications

Sickle cell disease: time for a closer look at treatment options?

Sickle cell disease: time for a closer look at treatment options?

Lung Function and Inflammation in Adult Sickle Cell Disease in a West Indian Sickle Cell Cohort

Glomerular filtration rate is altered in children with sickle cell disease: a comparison between Hb SS and Hb SC

Contact Info

Product

Resources

About