C-Path launches consortium to support medical product development in sickle cell disease

(Critical Path Institute (C-Path)) C-Path) today announced the launch of the Critical Path for Sickle Cell Disease (CP-SCD) Consortium to support collaboration and regulatory endorsement of new medical product development tools for sickle cell disease. These tools will help to optimize and de-risk clinical trials to increase efficiency in developing and delivering safe, effective treatments for people living with sickle cell disease.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news

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We describe the most highly recommended generic and disease-specific PRO tools in SCD and discuss the challenges of incorporating them in clinical practice. EXPERT OPINION: PRO measures are essential to incorporate into SCD clinical trials either as primary or secondary outcomes. The use of PRO measures in SCD facilitates a patient-centered approach, which is likely to lead to improved outcomes. Significant challenges remain in adapting PRO tools to routine clinical use and in developing countries. PMID: 33034214 [PubMed - as supplied by publisher]
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
Publication date: Available online 9 October 2020Source: Blood Cells, Molecules, and DiseasesAuthor(s): Melissa Azul, Surbhi Shah, Sarah Williams, Gregory M. Vercellotti, Alexander A. Boucher
Source: Blood Cells, Molecules, and Diseases - Category: Hematology Source Type: research
Publication date: Available online 8 October 2020Source: Hematology/Oncology and Stem Cell TherapyAuthor(s): M.M. Sulaiman, A.I. Ladu, A.M. Abba, A.A. Bukar
Source: Hematology Oncology and Stem Cell Therapy - Category: Cancer & Oncology Source Type: research
Condition:   Sickle Cell Anemia Intervention:   Drug: Voxelotor Sponsors:   Elizabeth Yang;   Mednax Center for Research, Education, Quality and Safety;   University of California, San Francisco;   Global Blood Therapeutics Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
AbstractSickle cell disease (SCD) is a genetic disorder predominantly affecting people of African descent and is associated with significant morbidity and mortality. To improve SCD outcomes, the National Heart Lung and Blood Institute funded eight centers to participate in the SCD Implementation Consortium. Sites were required to each recruit 300 individuals with SCD, over 20  months. We aim to describe recruitment strategies and challenges encountered. Participants aged 15–45 years with confirmed diagnosis of SCD were eligible. Descriptive statistics were used to analyze the effectiveness of each recruitme...
Source: Journal of Immigrant and Minority Health - Category: International Medicine & Public Health Source Type: research
ConclusionThe prevalence of RLS among this sample of  adult patients with sickle cell anemia was much higher than that previously reported for the general Turkish population. The presence of RLS was an important cause of sleep disturbance in these adult patients with SCA.
Source: Sleep and Breathing - Category: Respiratory Medicine Source Type: research
CONCLUSION: The prevalence of RLS among this sample of adult patients with sickle cell anemia was much higher than that previously reported for the general Turkish population. The presence of RLS was an important cause of sleep disturbance in these adult patients with SCA. PMID: 33029692 [PubMed - as supplied by publisher]
Source: Sleep and Breathing - Category: Sleep Medicine Authors: Tags: Sleep Breath Source Type: research
Conclusion: The risk of pregnancy in patients with homozygous sickle cell anemia remains high, on both the maternal and fetal sides. PMID: 33014460 [PubMed]
Source: Anemia - Category: Hematology Tags: Anemia Source Type: research
Abstract Total pancreatectomy with islet autotransplantation is a complex surgical approach for acute recurrent or chronic pancreatitis that frequently triggers extreme thrombocytosis (platelets ≥ 1000 × 109 /L). Thrombocytosis can be prothrombotic, so cytoreductive hydroxyurea is often initiated after this surgery; however, optimal dosing strategy and efficacy are unknown. This prospective pilot study characterized the pharmacokinetics of hydroxyurea after this procedure in children. It also compared them with previously published pediatric parameters in sickle cell anemia (SCA), the disease in which ped...
Source: The Journal of Clinical Pharmacology - Category: Drugs & Pharmacology Authors: Tags: J Clin Pharmacol Source Type: research
(University of Illinois at Chicago) Researchers at the University of Illinois Chicago will lead a $7.1 million, five-year national study to determine the effectiveness of acupuncture and guided relaxation for people with chronic pain from sickle cell disease. The National Institutes of Health grant is part of its Helping to End Addiction Long-term Initiative. The initiative aims to improve prevention and treatment strategies for opioid misuse and addiction and to enhance pain management.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
More News: Clinical Trials | International Medicine & Public Health | Sickle Cell Anemia