scholarly journals Effect ofN-acetylcysteine on pain in daily life in patients with sickle cell disease: a randomised clinical trial

2017 ◽  
Vol 182 (3) ◽  
pp. 444-448 ◽  
Author(s):  
Joep W. R. Sins ◽  
Karin Fijnvandraat ◽  
Anita W. Rijneveld ◽  
Martine B. Boom ◽  
Jean-Louis H. Kerkhoffs ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Daily Life ◽  
Randomised Clinical Trial ◽  
Cell Disease

Higher oxygen saturation with hydroxyurea in paediatric sickle cell disease

2019 ◽  
Vol 105 (6) ◽  
pp. 575-579 ◽  
Author(s):  
Lisa van Geyzel ◽  
Michele Arigliani ◽  
Baba Inusa ◽  
Bethany Singh ◽  
Wanda Kozlowska ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Sickle Cell Disease ◽  
Life Expectancy ◽  
Oxygen Saturation ◽  
Sickle Cell ◽  
Randomised Clinical Trial ◽  
Cell Disease ◽  
Respiratory Problems ◽  
Sleep Studies ◽  
Before And After

IntroductionSickle cell disease (SCD) is one of the most common inherited diseases worldwide. It is associated with lifelong morbidity and reduced life expectancy. Hydroxyurea (HU) has been shown to reduce the frequency and severity of vaso-occlusive episodes in SCD. Hypoxaemia and intermittent nocturnal oxygen desaturations occur frequently in children with SCD and contribute to the associated morbidity, including risk of cerebrovascular disease.ObjectiveTo evaluate the effect of HU on oxygen saturation (SpO2) overnight and on daytime SpO2 spot checks in children with SCD.MethodsA retrospective review of children with SCD and respiratory problems who attended two UK tertiary sickle respiratory clinics and were treated with HU. Longitudinal data were collected from 2 years prior and up to 3 years after the commencement of HU.ResultsForty-three children, 23 males (53%) with a median age of 9 (range 1.8–18) years were included. In the 21 children who had comparable sleep studies before and after starting HU, mean SpO2 was higher (95.2% from 93.5%, p=0.01) and nadir SpO2 was higher (87.2% from 84.3%, p=0.009) when taking HU. In 32 of the children, spot daytime oxygen saturations were also higher (96.3% from 93.5%, p=0.001).ConclusionChildren with SCD had higher oxygen saturation overnight and on daytime spot checks after starting HU. These data suggest HU may be helpful for treating persistent hypoxaemia in children with SCD pending more evidence from a randomised clinical trial.

scholarly journals End points for sickle cell disease clinical trials: patient-reported outcomes, pain, and the brain

2019 ◽  
Vol 3 (23) ◽  
pp. 3982-4001 ◽  
Author(s):  
Ann T. Farrell ◽  
Julie Panepinto ◽  
C. Patrick Carroll ◽  
Deepika S. Darbari ◽  
Ankit A. Desai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Patient Reported Outcomes ◽  
Cell Disease ◽  
End Points ◽  
Additional Clinical Trial ◽  
Patient Reported ◽  
The Brain

Abstract To address the global burden of sickle cell disease (SCD) 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 outcomes (PROs), pain (non-PROs), the brain, end-organ considerations, biomarkers, measurement of cure, and low-resource settings. This article presents the findings and recommendations of the PROs, pain, and brain panels, as well as relevant findings and recommendations from the biomarkers panel. The panels identify end points, where there were supporting data, to use in clinical trials of SCD. In addition, the panels discuss where further research is needed to support the development and validation of additional clinical trial end points.

scholarly journals Implementing a standard-of-care clinic for stroke prevention in children with sickle cell disease in Nigeria: a feasible strategy outside a clinical trial setting

2017 ◽  
Vol 1 (Suppl) ◽  
pp. 23-25
Author(s):  
Najibah A. Galadanci ◽  
Shehu U. Abdullahi ◽  
Leah D. Vance ◽  
Musa A. Tabari ◽  
Shehi Abubakar ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stroke Prevention ◽  
Standard Of Care ◽  
Cell Disease ◽  
Care Clinic ◽  
Clinical Trial Setting ◽  
Trial Setting

Clinical Trial Decision Making in Pediatric Sickle Cell Disease

2015 ◽  
Vol 37 (6) ◽  
pp. 415-422 ◽  
Author(s):  
Chavis A. Patterson ◽  
Veronica Chavez ◽  
Valerie Mondestin ◽  
Janet Deatrick ◽  
Yimei Li ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Decision Making ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Cell Disease ◽  
Pediatric Sickle Cell Disease

scholarly journals Clinical trial considerations in sickle cell disease: patient-reported outcomes, data elements, and the stakeholder engagement framework

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 196-205
Author(s):  
Sherif M. Badawy
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Stakeholder Engagement ◽  
Patient Reported Outcomes ◽  
Outcomes Assessment ◽  
Cell Disease ◽  
Patient Centered ◽  
Patient Reported

Abstract Patients with sickle cell disease (SCD) have significant impairment in their quality of life across the life span as a consequence of serious disease burden with several SCD-related complications. A number of disease-modifying therapies are currently available, yet long-term clinical benefits in real-world settings remain unclear. Over the past few years, a number of important initiatives have been launched to optimize clinical trials in SCD in different ways, including: (1) established panels through a partnership between the American Society of Hematology (ASH) and the US Food and Drug Administration; (2) the ASH Research Collaborative SCD Clinical Trials Network; (3) the PhenX Toolkit (consensus measures for Phenotypes and eXposures) in SCD; and (4) the Cure Sickle Cell Initiative, led by the National Heart, Lung, and Blood Institute. Electronic patient-reported outcomes assessment is highly recommended, and patient-reported outcomes (PROs) should be evaluated in all SCD trials and reported using Standard Protocol Items Recommendations for Interventional Trials guidelines. Patient-centered outcomes research (PCOR) approaches and meaningful stakeholder engagement throughout the process have the potential to optimize the execution and success of clinical trials in SCD with considerable financial value. This article reviews several clinical trial considerations in SCD related to study design and outcomes assessment as informed by recent initiatives as well as patient-centered research approaches and stakeholder engagement. A proposed hematology stakeholder-engagement framework for clinical trials is also discussed.

scholarly journals Comparative Effectiveness of a Web-Based Patient Decision Aid for Therapeutic Options for Sickle Cell Disease: Randomized Controlled Trial

10.2196/14462 ◽  
2019 ◽  
Vol 21 (12) ◽  
pp. e14462
Author(s):  
Lakshmanan Krishnamurti ◽  
Diana Ross ◽  
Cynthia Sinha ◽  
Traci Leong ◽  
Namita Bakshi ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Decision Making ◽  
Sickle Cell Disease ◽  
Randomized Clinical Trial ◽  
Sickle Cell ◽  
Decision Aid ◽  
Care Providers ◽  
Patient Decision Aid ◽  
Cell Disease ◽  
Patient Decision

Background Hydroxyurea, chronic blood transfusions, and bone marrow transplantation are efficacious, disease-modifying therapies for sickle cell disease but involve complex risk-benefit trade-offs and decisional dilemma compounded by the lack of comparative studies. A patient decision aid can inform patients about their treatment options, the associated risks and benefits, help them clarify their values, and allow them to participate in medical decision making. Objective The objective of this study was to develop a literacy-sensitive Web-based patient decision aid based on the Ottawa decision support framework, and through a randomized clinical trial estimate the effectiveness of the patient decision aid in improving patient knowledge and their involvement in decision making. Methods We conducted population decisional needs assessments in a nationwide sample of patients, caregivers, community advocates, policy makers, and health care providers using qualitative interviews to identify decisional conflict, knowledge and expectations, values, support and resources, decision types, timing, stages and learning, and personal clinical characteristics. Interview transcripts were coded using QSR NVivo 10. Alpha testing of the patient decision aid prototype was done to establish usability and the accuracy of the information it conveyed, and then was followed by iterative cycles of beta testing. We conducted a randomized clinical trial of adults and of caregivers of pediatric patients to evaluate the efficacy of the patient decision aid. Results In a decisional needs assessment, 223 stakeholders described their preferences, helping to guide the development of the patient decision aid, which then underwent alpha testing by 30 patients and 38 health care providers and iterative cycles of beta testing by 87 stakeholders. In a randomized clinical trial, 120 participants were assigned to either the patient decision aid or standard care (SC) arm. Qualitative interviews revealed high levels of usability, acceptability, and utility of the patient decision aid in education, values clarification, and preparation for decision making. On the acceptability survey, 72% (86/120) of participants rated the patient decision aid as good or excellent. Participants on the patient decision aid arm compared to the SC arm demonstrated a statistically significant improvement in decisional self-efficacy (P=.05) and a reduction in the informed sub-score of decisional conflict (P=.003) at 3 months, with an improvement in preparation for decision making (P<.001) at 6 months. However, there was no improvement in terms of the change in knowledge, the total or other domain scores of decisional conflicts, or decisional self-efficacies at 6 months. The large amount of missing data from survey completion limited our ability to draw conclusions about the effectiveness of the patient decision aid. The patient decision aid met 61 of 62 benchmarks of the international patient decision aid collaboration standards for content, development process, and efficacy. Conclusions We have developed a patient decision aid for sickle cell disease with extensive input from stakeholders and in a randomized clinical trial demonstrated its acceptability and utility in education and decision making. We were unable to demonstrate its effectiveness in improving patient knowledge and involvement in decision making. Trial Registration ClinicalTrials.gov NCT03224429; https://clinicaltrials.gov/ct2/show/NCT03224429 and ClinicalTrials.gov NCT02326597; https://clinicaltrials.gov/ct2/show/NCT02326597

Barriers to Deferoxamine Adherence for Adults with Sickle Cell Disease.

Blood ◽  
2004 ◽  
Vol 104 (11) ◽  
pp. 3760-3760 ◽  
Author(s):  
Marsha Treadwell ◽  
Jennifer Sung ◽  
Eileen Murray ◽  
Robert Hagar ◽  
Kimberly Major ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Sickle Cell Disease ◽  
Adverse Effects ◽  
Iron Overload ◽  
Sickle Cell ◽  
Serum Ferritin ◽  
Chelation Therapy ◽  
Clinical Care ◽  
Cell Disease ◽  
Transfusion Therapy

Abstract Background: The barriers to adherence with chelation therapy for chronically transfused and iron overloaded patients with sickle cell disease (SCD) have been described only anecdotally. Objectives: To describe barriers to home deferoxamine (DFO) administration adherence among adults with SCD. It was hypothesized that barriers would include limited patient education on the importance of chelation and perceived aversiveness of the regimen. Methods: Medical records were reviewed for 189 adult patients seen at a comprehensive sickle cell center. Patients with transfusion induced hemosiderosis, defined as a serum ferritin ≥ 1500 ng/ml, were administered a four item interview asking if iron overload had ever been discussed with them; if they had been informed they were iron over loaded; if chelation therapy had been offered; and if not currently home chelating, why not. Patients not interviewed were deceased (3); unavailable (10); or declined (3). A study coordinator who did not provide clinical care conducted the interviews. Results: 54 of the 189 patients (29%) had a history of intermittent or chronic transfusion, or pheresis. 45 of these patients were iron overloaded. 29 of these patients agreed to complete the interview; 22 (76%) were female. Average age was 41.5 years (range 22.4 – 58.4 years) and average serum ferritin was 4240.8 (range 1547 – 9420). 23 of the 29 patients (79%) reported that their physician or nurse had discussed iron overload and chelation with them. 16 of these (55%) reported that they were currently receiving home DFO therapy. Reasons given for not administering home DFO included: Reason Number (%) “Don’t want to stick self” 3 (23) No longer being transfused or being exchanged 3 (23) Awaiting clinical trial for oral chelator 2 (15) Home situation too complex 2 (15) Don’t want to (no further explanation) 2 (15) Too many adverse effects 1 (8) Discussion: Life threatening levels of iron overload were observed in intermittently transfused adult sickle cell patients. Contrary to expectations, iron overload and its treatment had been discussed with most patients. However, just over half were currently chelating at home. Toxicity of DFO and misunderstanding that iron overload is no longer a problem if chronic transfusion therapy stops are the most common reasons for non-compliance. Repeated patient counseling are essential in order to prevent progressive iron toxicity in sickle cell disease. Reason Number (%) “Don’t want to stick self” 3 (23) No longer being transfused or being exchanged 3 (23) Awaiting clinical trial for oral chelator 2 (15) Home situation too complex 2 (15) Don’t want to (no further explanation) 2 (15) Too many adverse effects 1 (8)

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