Tapered oral dexamethasone for the acute chest syndrome of sickle cell disease

Quinn, Charles T.; Stuart, Marie J.; Kesler, Karen; Ataga, Kenneth I.; Wang, Winfred C.; Styles, Lori; Smith‐Whitley, Kim; Wun, Ted; Raj, Ashok; Hsu, Lewis L.; Krishnan, Suba; Kuypers, Frans A.; Setty, Yamaja; Rhee, Sang Youl; Key, Nigel S.; Buchanan, George R.

doi:10.1111/j.1365-2141.2011.08827.x

Cited by 34 publications

(37 citation statements)

References 16 publications

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“…Corticosteroids can shorten the duration and severity of ACS but can also precipitate “rebound” painful events. 12,13 It is reasonable to use corticosteroids for patients with asthma when it is otherwise indicated (eg, an exacerbation of asthma with ACS) or for rapidly progressive or severe ACS with the intent of preventing or limiting mechanical ventilation.…”

Section: Clinical Scenarios and Complications Of Scdmentioning

confidence: 99%

Sickle Cell Disease in Childhood

Quinn

2013

Pediatric Clinics of North America

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Section: Clinical Scenarios and Complications Of Scdmentioning

confidence: 99%

Sickle Cell Disease in Childhood

Quinn

2013

Pediatric Clinics of North America

Self Cite

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“…From the investigator perspective, a few sickle cell studies that were terminated early performed an analysis of their obstacles in trial conduct, which included limitations in research infrastructure or funding for dedicated research staff, overstated expected enrollment by investigators during trial planning, and difficulty enrolling participants based on strict eligibility requirements, including both time constraints and potential bias in not approaching all eligible participants. 22, 25, 35 As a rare disease, it is vital that investigators continue to focus both on improving participant engagement and limiting obstacles in trial design and compliance. This design and possibly pilot testing should be conducted during the planning phase rather than during the conduct of the trial, as ineffective enrollment once a trial has been funded may take too long to correct and can lead to early termination.…”

Section: Discussionmentioning

confidence: 99%

Systematic review of interventional sickle cell trials registered in ClinicalTrials.gov

et al. 2015

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Background/Aims The registry clinicaltrials.gov was created to provide investigators and patients an accessible database of relevant clinical trials. Methods To understand the state of sickle cell disease (SCD) clinical trials, a comprehensive review of all 174 “closed”, “interventional” sickle cell trials registered at clinicaltrials.gov was completed January 2015. Results The majority of registered SCD clinical trials listed an academic center as the primary sponsor and were an early phase trial. The primary outcome for SCD trials focused on pain (23%), bone marrow transplant (13%), hydroxyurea (8%), iron overload (8%), and pulmonary hypertension (8%). Fifty two trials were listed as terminated or withdrawn including 25 (14% of all trials) terminated for failure to enroll participants. At the time of this review, only 19 trials uploaded results and 29 trials uploaded a manuscript in the clinicaltrials.gov database. A systematic review of pubmed.gov revealed that only 35% of sickle cell studies completed prior to 2014 resulted in an identified manuscript. In comparison, of 80 thalassemia trials registered in clinicaltrials.gov four acknowledged failure to enroll participants as a reason for trial termination or withdrawal and 48 trials (60%) completed prior to 2014 resulted in a currently identified manuscript. Conclusion Clinicaltrials.gov can be an important database for investigators and patients with sickle cell disease to understand the current available research trials. To enhance the validity of the website, investigators must update their trial results and upload trial manuscripts into the database. This study, for the first time, quantifies outcomes of SCD trials and provides support to the belief that barriers exist to successful completion, publication, and dissemination of sickle cell trial results.

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“…The role of corticosteroids, specifically dexamethasone or prednisone, in the management of STA is controversial, since there is a significant variability in its efficacy [56]. Studies indicate that there is a reduction in the time of hospitalization of patients who use corticosteroids, however, concomitantly other studies show a high rate of readmission of pain 72 h after therapy [31,56,57]. Another central issue is the high risk of corticosteroidinduced fat embolism in patients with SCD, and it is not recommended to administer corticosteroid in these patients [54].…”

Section: Corticosteroidsmentioning

confidence: 99%

Therapeutic Approaches of Acute Thoracic Syndrome in Patients with Falciform Disease

Ac¹,

Costa²,

Tec³

et al. 2017

J Blood Lymph

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Sickle cell disease (SCD) is an autosomal recessive inheritance disorder that affects the beta-globin gene and results in the replacement of the amino acid glutamic acid by valine in the β chain of the hemoglobin molecule, producing erythrocytes with defective forms and functions. A range of pathological conditions is associated with SCD, however, one in particular stands out by gravity. Acute thoracic syndrome (STA), characterized by the presence of pulmonary infiltrates associated with a clinical symptom such as chest pain, cough, wheezing, tachypnea and fever, is considered a leading cause of death in patients with sickle cell disease. Early diagnosis and introduction of an effective approach to complication are needed to improve outcomes and minimize associated morbidity and mortality. Risk factors associated with STA in the SCDAbdominal surgery for splenectomy and cholecystectomy [19] Hypersensitivity [20,21] Smoking [20,21] Asthma [22] Infections [9,[23][24][25] Post-Surgical Hypoventilation [9,[23][24][25] Fatty Embolism [9,[23][24][25]

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Tapered oral dexamethasone for the acute chest syndrome of sickle cell disease

Cited by 34 publications

References 16 publications

Sickle Cell Disease in Childhood

Sickle Cell Disease in Childhood

Systematic review of interventional sickle cell trials registered in ClinicalTrials.gov

Therapeutic Approaches of Acute Thoracic Syndrome in Patients with Falciform Disease

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