A pilot study of electronic directly observed therapy to improve hydroxyurea adherence in pediatric patients with sickle-cell disease

2014 ◽  
Vol 61 (6) ◽  
pp. 1068-1073 ◽  
Author(s):  
Susan E. Creary ◽  
Mark T. Gladwin ◽  
Melissa Byrne ◽  
Mariana Hildesheim ◽  
Lakshmanan Krishnamurti
Keyword(s):  
Pilot Study ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Directly Observed Therapy ◽  
Cell Disease

Computerized cognitive training in pediatric sickle cell disease: A randomized controlled pilot study.

2020 ◽  
Vol 8 (4) ◽  
pp. 390-401 ◽  
Author(s):  
Taryn M. Allen ◽  
Lindsay M. Anderson ◽  
Samuel M. Brotkin ◽  
Jennifer A. Rothman ◽  
Melanie J. Bonner
Keyword(s):  
Pilot Study ◽  
Sickle Cell Disease ◽  
Cognitive Training ◽  
Sickle Cell ◽  
Cell Disease ◽  
Computerized Cognitive Training ◽  
Randomized Controlled ◽  
Pediatric Sickle Cell Disease

Surfactant protein D as a marker for pulmonary complications in pediatric patients with sickle cell disease: Relation to lung function tests

2019 ◽  
Vol 54 (5) ◽  
pp. 610-619 ◽  
Author(s):  
Azza A. Tantawy ◽  
Amira A. Adly ◽  
Fatma S. E. Ebeid ◽  
Eman A. Ismail ◽  
Mahitab M. Hussein ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Lung Function ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Surfactant Protein ◽  
Pulmonary Complications ◽  
Surfactant Protein D ◽  
Lung Function Tests ◽  
Cell Disease ◽  
Function Tests

scholarly journals VEGF Promoter Region 18-bp Insertion-Deletion Polymorphism in Sickle Cell Disease Patients with Microalbuminuria: A Pilot Study

2018 ◽  
Vol 35 (2) ◽  
pp. 278-283
Author(s):  
Dnyanesh B. Amle ◽  
Rachana L. Patnayak ◽  
Varsha Verma ◽  
Gajendra Kumar Singh ◽  
Vijaylakshmi Jain ◽  
...  
Keyword(s):  
Pilot Study ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Promoter Region ◽  
Cell Disease ◽  
Deletion Polymorphism

scholarly journals A pilot study of the effect of atorvastatin on endothelial function and albuminuria in sickle cell disease

2019 ◽  
Vol 94 (11) ◽  
Author(s):  
Kenneth I. Ataga ◽  
David Wichlan ◽  
Laila Elsherif ◽  
Vimal K. Derebail ◽  
Adane F. Wogu ◽  
...  
Keyword(s):  
Pilot Study ◽  
Sickle Cell Disease ◽  
Endothelial Function ◽  
Sickle Cell ◽  
Cell Disease

scholarly journals Efficacy of Allogeneic Hematopoietic Cell Transplantation (HCT) in Sickle Cell Disease: Results of a Systematic Review/Meta-Analysis

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2106-2106
Author(s):  
Madiha Iqbal ◽  
Tea Reljic ◽  
Ernesto Ayala ◽  
Hemant S. Murthy ◽  
Ambuj Kumar ◽  
...  
Keyword(s):  
Systematic Review ◽  
Sickle Cell Disease ◽  
Clinical Outcomes ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Medical Literature ◽  
Meta Analysis ◽  
Adult Patients ◽  
Cell Disease ◽  
Allogeneic Hct

Background: Sickle cell disease (SCD) is an inherited hemoglobinopathy which affects over 300,000 children born each year worldwide. In spite of improvement in supportive care in recent years, there is still a lack of effective treatment options. SCD leads to debilitating and cyclic episodes of erythrocyte sickling with progressive organ injury, contributing to lifetime morbidity and shortened life expectancy. Allogeneic HCT (allo-HCT) is a potentially curative therapy for SCD because engraftment is associated with resolution of the clinical phenotype of the disease and abrogation of its complications. Medical literature on allo-HCT for SCD is largely limited to children. Recent studies have evaluated the efficacy of allo-HCT in the adult population. Here, we conduct a systematic review/meta-analysis to assess the totality of evidence pertaining to the efficacy (or lack thereof) of allo-HCT in children and adults. Materials and methods: We performed a comprehensive search of the medical literature using PubMed/Medline, EMBASE and Cochrane library on July 3rd, 2019. We extracted data on clinical outcomes related to benefits (overall [OS] and disease free/event free survival [EFS/DFS]) and harms (non-relapse mortality [NRM] and graft failure [GF]), independently by two authors. Our search strategy identified 1001 references but only 30 studies (n= 1995 patients) were included in this systematic review/meta-analysis. We also performed a sub analysis on clinical outcomes for studies that included only pediatric patients (defined as <18 years) and those in patients ≥18 years of age. Results: Median age for patients enrolled in all the studies was at 10 years. Recurrent veno-occlusive crises represented the most common indication for allo-HCT followed by acute chest syndrome and stroke; nevertheless, most patients had more than one indication. Matched related donors (MRD) were the most common donor source (93%). Bone marrow was the most common source of hematopoietic stem cells (77%). Majority of patients underwent conditioning with myeloablative regimens (77%). Pooled OS rates (n=29 studies, 1681 patients) after allogeneic HCT was 95% (95%CI=93-96%) with low heterogeneity (I2=6.4%) among included studies (Figure 1). Pooled EFS/DFS rates (n=29 studies, 1894 patients) post-allografting was 90% (95%CI=87-93%) with moderate heterogeneity (I2=54%). Pooled NRM rates from 30 studies (1995 patients) was 4% (95%CI=2-6%) with low heterogeneity (I2=29.4%). Pooled GF rates from 28 studies (1851 patients) was 4% (95%CI=2-6%) with moderate heterogeneity (I2=55%). A subset analysis specifically for pediatric patients (n= 11 studies, 1009 patients, median age at 9.7 years) showed a pooled OS rate of 96% (95%CI=94-97%) with low heterogeneity (I2=0%); and for adult patients (n=3 studies, 51 patients, median age at 33.4 years) the pooled OS was 94% (95%CI=80-100%) with moderate heterogeneity (I2=52%). Pooled EFS/DFS for pediatric patients (n= 11 studies, 1009 patients) was at 89 %( 95%CI=84-93%) with moderate heterogeneity (I2=55.1%); and for adult patients (n=2 studies, 30 patients) was at 95% (95%CI=83-100%) with high heterogeneity (I2=96.5%). Pooled NRM from 10 studies with pediatric patients (281 patients) was at 6 % (95%CI=3-10%) with low heterogeneity (I2=0%); and from 3 studies with adult patients (51 patients) was at 1% (95%CI=0-7%) with low heterogeneity (I2=15.1%). Pooled GF from 10 studies with pediatric patients (281 patients) was at 3 % (95%CI=1-7%) with moderate heterogeneity (I2=40%); and from 2 studies with adult patients (30 patients) was at 5% (95%CI=0-17%) with high heterogeneity (I2=95.4%). Conclusions: The results of our systematic review/meta-analysis show excellent OS, EFS/DFS in children and adults undergoing allo-HCT with pooled OS rates exceeding 90%. The main limitation to offering an allo-HCT in SCD remains the availability of a suitable donor as 85% of patients meeting criteria do not have a MRD. We anticipate that with emergence of haploidentical transplantation the number of allo-HCT will increase in the future. GF remains a significant concern in this population and future studies should focus on novel immune suppression strategies to help reduce GF. Disclosures Kharfan-Dabaja: Pharmacyclics: Consultancy; Daiichi Sankyo: Consultancy.

scholarly journals Sickle Cell Disease: An Overview of Oral Health Considerations for Pediatric Patients

2021 ◽  
Vol 2 (3) ◽  
pp. 9-17
Author(s):  
Dafni Eleftherou ◽  
Aristidis Arhakis ◽  
Sotiria Davidopoulou
Keyword(s):  
Oral Health ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Genetic Disorder ◽  
The Body ◽  
Common Finding ◽  
Cell Disease ◽  
Preventive Dental Care ◽  
Periodontal Inflammation

Aim: This literature review aims to update the evidence for orofacial manifestations and current treatment recommendations for children and adolescents with sickle cell disease. Background: Sickle cell disease is a frequent hemoglobinopathy and a life-threatening genetic disorder. The lifelong condition is characterized by chronic hemolytic anemia and vaso-occlusive crisis that may occur in a variable range of clinical presentations in different regions of the body, including the oral cavity. Review results: This review explored the most common orofacial alterations of pediatric patients with SCD. Dental caries is a common finding in SCD pediatric patients, especially in those who are socio-economically vulnerable. Moreover, malocclusions occur in high prevalence in SCD pediatric patients. Other oral health complications seen in SCD patients include periodontal inflammation, bone changes, infections, mental nerve neuropathy, facial overgrowth, delayed tooth eruption, dental anomalies, pulp necrosis, soft tissue alterations and salivary changes. Dental infections may trigger a vaso-occlusive crisis leading the patient to a higher probability on arriving in hospital emergency departments and in need for further hospital admission to deal with the correlated complications. Thus, preventive dental care and non-invasive dental procedures are the principal focus in SCD patients in order to avoid possible subsequent complications. Conclusion: The review showed that in pediatric patients with SCD the risk for orofacial manifestations and complications depends not only on the presence of SCD but also on other confounding factors such as oral hygiene, diet habits and social conditions. Moreover, more well-designed epidemiological studies are necessary to assess the real link between SCD disease and its impact on stomatognathic health.

scholarly journals CD209-336A/G promotor polymorphism and its clinical associations in sickle cell disease Egyptian Pediatric patients

2018 ◽  
Vol 11 (2) ◽  
pp. 75-81 ◽  
Author(s):  
Rasha Abdel-Raouf Afifi ◽  
Dina Kamal ◽  
Riham El. Sayed ◽  
Sherif M.M. Ekladious ◽  
Gehan H. Shaheen ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Cell Disease ◽  
Clinical Associations

Early Identification of Barriers and Facilitators to Self-Management Behaviors in Pediatric Patients With Sickle Cell Disease to Minimize Hematopoietic Cell Transplantation Complications

2018 ◽  
Vol 35 (3) ◽  
pp. 199-209 ◽  
Author(s):  
Ginny L. Schulz ◽  
Rebecca H. Foster ◽  
Valerie Kennedy Lang ◽  
Alison Towerman ◽  
Shalini Shenoy ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Hematopoietic Cell Transplantation ◽  
Hematopoietic Cell ◽  
Barriers And Facilitators ◽  
Self Management ◽  
Cell Disease ◽  
Management Behaviors

Hematopoietic cell transplantation (HCT) is an elective, curative treatment option for patients with sickle cell disease (SCD). Transplant requires extensive self-management behaviors to be successful. The purpose of this study was to describe potential barriers and facilitators to self-management in a group of pediatric patients with SCD prior to HCT and their medical outcomes post-HCT. A multiple case study approach was used to describe 4 pairs of transplant recipients grouped by age, donor type, and donor source. Each pair included a case with minimal and increased post-HCT complications. Complications included readmissions, graft-versus-host disease, systemic infections, and survival in the first year post-HCT. Variables were retrospectively collected and content analyzed to identify barriers and facilitators within and across pairs using existing self-management frameworks. While higher risk transplants experienced more complications, 3 of the 4 cases with increased complications had a larger number of modifiable barriers identified compared with those experiencing minimal complications. At least one modifiable barrier and multiple facilitators were identified in all cases. A standardized psychosocial assessment process with an established plan to mitigate barriers and promote facilitators to self-management is essential to optimize outcomes in patients with SCD undergoing elective HCT.

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