scholarly journals Disease-Specific Patient-Reported Outcome Instruments In Sickle Cell Disease: A Systematic Literature Review

2017 ◽  
Vol 20 (9) ◽  
pp. A561
Author(s):  
J Gupta ◽  
A Ghildiyal
Keyword(s):  
Sickle Cell Disease ◽  
Literature Review ◽  
Sickle Cell ◽  
Systematic Literature Review ◽  
Patient Reported Outcome ◽  
Cell Disease ◽  
Specific Patient ◽  
Patient Reported ◽  
Disease Specific

scholarly journals End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings

2019 ◽  
Vol 3 (23) ◽  
pp. 4002-4020 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
Ankit A. Desai ◽  
Adetola A. Kassim ◽  
Jeffrey Lebensburger ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcome ◽  
Cell Disease ◽  
End Points ◽  
Low Resource Settings ◽  
Low Resource ◽  
Patient Reported ◽  
The Us ◽  
American Society

Abstract To address the global burden of sickle cell disease and the need for novel therapies, the American Society of Hematology partnered with the US Food and Drug Administration to engage the work of 7 panels of clinicians, investigators, and patients to develop consensus recommendations for clinical trial end points. The panels conducted their work through literature reviews, assessment of available evidence, and expert judgment focusing on end points related to patient-reported outcome, pain (non–patient-reported outcomes), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the end-organ considerations, measurement of cure, and low-resource settings panels as well as relevant findings and recommendations from the biomarkers panel.

Sickle Cell Disease and Pulpal Necrosis: A Review of the Literature for the Primary Care Dentist

2014 ◽  
Vol 3 (1) ◽  
pp. 76-79 ◽  
Author(s):  
M S Basati
Keyword(s):  
Primary Care ◽  
Sickle Cell Disease ◽  
Literature Review ◽  
Sickle Cell ◽  
Alveolar Bone ◽  
Cell Disease ◽  
General Dental Practitioner ◽  
Patient Reported ◽  
Primary Care Practitioners ◽  
The Relationship

A case of difficult diagnosis of a patient with sickle cell disease and tooth pain fuelled a literature review by a primary care general dental practitioner. A literature search was conducted to review the relationship between sickle cell disease and pulpal necrosis. The results indicated statistically significant associations of sickle cell disease and tooth pain without any carious or traumatic pathology. It is important for the primary care dentist to be aware of the relationship between sickle cell disease and pulpal necrosis to prevent misdiagnosis and consequently mistreatment. Background A female patient, 49, of African-Carribean descent with sickle cell disease presented to the dental clinic with chronic pain. The tooth involved was an upper right first premolar free of caries and trauma. On a periapical radiograph the surrounding alveolar bone had large areas of ‘black banding’. The patient reported that large ‘black bands’ had also been seen on radiographs of her hip and shoulder and eventually the orthopaedic physician had come to the diagnosis of bone necrosis secondary to the patient's sickle cell disease. With this information, a provisional diagnosis of pulpal necrosis secondary to avascular necrosis was made, once all other possible diagnoses had been excluded. The premolar was extirpated and at a review appointment there was complete resolution of symptoms. The author performed a literature review because as a primary care dentist he had never come across this diagnosis before and noted that other primary care practitioners might be interested to learn about it.

scholarly journals Acceptability and Feasibility of a Disease-specific Patient Portal in Adolescents With Sickle Cell Disease

2019 ◽  
Vol 41 (7) ◽  
pp. 561-567 ◽  
Author(s):  
Katherine M. Kidwell ◽  
James Peugh ◽  
Emilie Westcott ◽  
Cara Nwankwo ◽  
Maria T. Britto ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Portal ◽  
Cell Disease ◽  
Specific Patient ◽  
Disease Specific

scholarly journals A Systematic Literature Review of the Burden of Ischemic Priapism in Patients with Sickle Cell Disease

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3467-3467
Author(s):  
Dave Nellesen ◽  
Sedge Lucas ◽  
Che-Rung Liu ◽  
Menaka Bhor ◽  
Jincy Paulose ◽  
...  
Keyword(s):  
Emergency Department ◽  
Erectile Dysfunction ◽  
Sickle Cell Disease ◽  
Literature Review ◽  
Sexual Function ◽  
Sickle Cell ◽  
Systematic Literature Review ◽  
Economic Burden ◽  
Emergency Department Visits ◽  
Cell Disease

INTRODUCTION: Ischemic or low-flow priapism is a serious condition characterized by a persistent, painful erection that lasts at least 4 hours or is unrelated to sexual stimulation. Both stuttering (repeated short episodes) and fulminant (long-lasting) priapism are prevalent in men and boys with sickle cell disease (SCD). Approximately 40% of patients with SCD will experience priapism during their lifetime. The objective of this study was to conduct a systematic literature review (SLR) to identify published studies reporting the burden associated with priapism in patients with SCD. METHODS: MEDLINE, Embase, Cochrane CENTRAL/CDSR and 14 congresses were searched from May 9-16, 2019. Results were screened against predefined criteria by two independent researchers. Studies assessing the clinical, humanistic, or economic burden of human patients with SCD and priapism (N≥15) were included. Outcomes of interest included priapism-related symptoms, treatment-related complications, quality of life (QoL), and economic burden. RESULTS: The literature search identified 1236 articles; 62 full-text studies were reviewed in detail and 34 studies were included in the analysis. Most studies were retrospective observational (21), others were prospective (7 observational, 6 interventional). Study size varied widely (range: 15-10,788 patients). Of 27 studies that reported mean or median patient age, the age statistic for patients with SCD and priapism ranged from 10 to 32. Overall, 19 studies reported at least one aspect of clinical burden. Thirteen reported the rate of erectile dysfunction or impotence, ranging from 0% to 56%. Eight reported treatment-related complications, including perceived penile scarring/deformity (19.4%) and small intrapenile hematoma (13.3%). Nineteen studies reported QoL outcomes. Eight reported physical outcomes, including a negative impact of SCD-related priapism on sleep quality and worse scores on periodic limb movement and apnea hypopnea indices. Seven reported measures of sexual function, including low rates of satisfactory sexual intercourse. One study reported a diminished Sexual Health Inventory for Men score in patients with active priapism, and another found a statistically significant relationship between stuttering priapism and premature ejaculation. Three studies reported measures of mental function, one of which outlined increased cognitive fatigue and impaired emotional functioning among children with priapism and another reported that 36% of patients had substantial to very extreme worry regarding episodes of priapism. Use of validated measures for QoL were limited: 3 studies assessed patients with the International Index of Erectile Function (IIEF) and 1 with the Priapism Impact Profile (PIP). No studies measuring the burden to caregivers of pediatric patients with priapism were identified. Eight studies reported the economic burden of priapism in patients with SCD, including rates of emergency department visits (4 studies: 56.5% sought hospital treatment - 26/46; 49.8% admission to the hospital out of emergency department visits - 5371/10,788; 50.4% admitted at the emergency department - 930/1844; 26% vs. 11% readmission vs no readmission - p <0.001) and hospital length of stay (4 studies: mean 3.38 days; mean 3.8 days; median 2 days; median 5 days in patients who received an exchange transfusion as a treatment for priapism.). Three studies reported direct treatment costs, ranging from a mean of $4496 (2015 USD) for patients who did not undergo penile operations to a mean of $19,670 (2013 USD) total adjusted hospital costs for patients receiving both transfusions and urologic procedures. CONCLUSIONS: This SLR identified 34 studies reporting the burden of priapism in patients with SCD. No RCTs were identified. The use of comprehensive or validated measures of humanistic burden was limited. Manifestations of priapism are wide-ranging, including painful treatment-related complications, impaired sleep, and diminished physical, mental and sexual function. Patients with priapism and SCD are more likely to visit the emergency department and experience longer hospital stays compared to SCD patients without priapism. Together, the evidence identified in this SLR indicates that priapism not only can result in erectile dysfunction, but can impair multiple dimensions of a patient's physical and mental health. Disclosures Nellesen: Analysis Group, Inc.: Consultancy. Lucas:Analysis Group, Inc.: Consultancy. Liu:Analysis Group, Inc.: Consultancy. Bhor:Novartis: Employment, Equity Ownership. Paulose:Novartis Pharmaceuticals Corporation: Employment.

scholarly journals A systematic literature review of frequency of vaso-occlusive crises in sickle cell disease

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Ahmar U. Zaidi ◽  
Alexander K. Glaros ◽  
Soyon Lee ◽  
Taiji Wang ◽  
Rhea Bhojwani ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Literature Review ◽  
Sickle Cell ◽  
Systematic Literature Review ◽  
Real World ◽  
Outpatient Setting ◽  
Cell Disease ◽  
Published Evidence ◽  
Significant Burden ◽  
At Home

Abstract Background and purpose Sickle cell disease (SCD) is a collection of rare inherited blood disorders affecting approximately 100,000 people in the U.S. and 20–25 million people globally. Individuals with SCD experience recurrent episodes of severe and unpredictable pain that are caused by vaso-occlusive crises (VOCs), a hallmark of the disease. VOCs are the primary cause of hospitalization in SCD, result in missed workdays and school days, and decrease quality of life (QoL). Although VOCs cause significant burden in the lives of individuals with SCD, there is no synthesis on the frequency of VOCs in the real world. This systematic literature review sought to identify literature describing the frequency of VOCs experienced by individuals with SCD in real-world settings. Methods MEDLINE and 6 congresses were searched (date range: January 1, 2000 to June 30, 2020). Studies were reviewed independently by two researchers. Studies assessing frequency or prevalence of VOCs or VOC-related outcomes were included. Results Of 1438 studies identified in the search, 52 met pre-specified inclusion and exclusion criteria. Reported frequency of VOCs varied widely ranging from a mean or median of 0 VOCs/year to 18.2 VOCs/year. The proportion of patients experiencing ≥ 3 VOCs/year ranged from 4 to 67% and the proportion of patients experiencing ≥ 5 VOCs/year ranged from 18 to 59%. Measures of VOC severity were limited, with 13 studies considering frequency of complicated VOCs and only 1 study reporting duration of VOC episodes. Conclusions This is the first study to systematically assess published evidence pertaining to VOCs in real-world settings. Reported VOC frequency in real-world settings varied widely, with a majority of studies only considering VOCs managed in an inpatient or outpatient setting. Studies that considered VOCs managed at home reported a higher frequency of VOCs, suggesting that many studies may underestimate the frequency of VOCs. This systematic literature review (SLR) highlights the need for consistent reporting of (1) self-reported VOCs, including those managed at home, (2) definitions of VOCs, (3) complicated VOCs, and (4) duration of VOC episodes in literature.

Microsurgery in the Sickle Cell Trait Population: Is It Actually Safe?

2020 ◽  
pp. 1-2
Author(s):  
Michael Alperovich ◽  
Eric Park ◽  
Michael Alperovich ◽  
Omar Allam ◽  
Paul Abraham
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Free Flap ◽  
Near Infrared ◽  
Sickle Cell Trait ◽  
Cell Disease ◽  
Flap Transfer ◽  
Free Flap Transfer ◽  
Patient Reported ◽  
Deep Inferior Epigastric Perforator

Although sickle cell disease has long been viewed as a contraindication to free flap transfer, little data exist evaluating complications of microsurgical procedures in the sickle cell trait patient. Reported is the case of a 55-year-old woman with sickle cell trait who underwent a deep inferior epigastric perforator (DIEP) microvascular free flap following mastectomy. The flap developed signs of venous congestion on postoperative day two but was found to have patent arterial and venous anastomoses upon exploration in the operating room. On near-infrared indocyanine green angiography, poor vascular flow was noted despite patent anastomoses and strong cutaneous arterial Doppler signals. Intrinsic microvascular compromise or sickling remains a risk in the sickle cell trait population as it does for the sickle cell disease population. Just like in sickle cell disease patients, special care should be taken to optimize anticoagulation and minimize ischemia-induced sickling for patients with sickle cell trait undergoing microsurgery.

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