NIH announces $1 million prize competition to target global disease diagnostics
Bill & Melinda Gates Foundation to offer supplementary support for designs that can be developed into products on a rapid timeframe. https://www.nih.gov/news-events/news-releases/nih-announces-1-million-prize-competition-target-global-disease-diagnosticsThe National Institutes of Health has launched a $1 million Technology Accelerator Challenge to spur the design and development of non-invasive, handheld, digital technologies to detect, diagnose and guide therapies for diseases with high global and public health impact. The challenge is focused on sickle cell disease, malaria and anemia and is led by NIH’s National Institute of Biomedical Imaging and Bioengineering (NIBIB). The Bill & Melinda Gates Foundation is cooperating with NIH to help accelerate the transformation of design concepts into products for low-resource settings. NIH will award up to $500,000 for a top finalist and smaller awards to approximately five semi-finalists. The Gates Foundation will separately review winners and honorable mentions and consider them for follow-on support. This may include a grant of up to $500,000 and/or consultations, partnerships for clinical data collection, software development, scale-up, and manufacturing. “Bioengineers are pioneering the development of cutting-edge, cost-effective, mobile and point-of-care technologies,” said NIBIB Director Bruce Tromberg, Ph.D. “This challenge is an exciting way to engage and inspire our community to address an urgent health care need.”Accessible diagnostic tools are essential for providing treatments and cures for some of the world’s highest-burden diseases. While diagnostics currently exist for sickle cell disease, malaria and anemia, they can be challenging to deliver in low-resource settings, particularly at the population level, due to cost, invasiveness and the expertise required to administer the tests. The current challenge is designed to stimulate the development of a platform technology that could be used to rapidly screen large populations as well as provide physicians with a practical tool for optimizing therapy in individual patients. “While this challenge is not constrained to any specific technology, the inspiration for it comes from the widespread availability of mobile phones and the potential for mobile phone-linked sensor technologies to non-invasively detect changes in the blood and blood vessels associated with these treatable diseases,” Tromberg said.
For low-resource settings, diagnostics would ideally be portable, self-contained, low-cost, adaptable to multiple diseases, and able to integrate information about the patient and the environment in interpreting the test result.“New diagnostic tools could address a major burden of disease in low- and middle-income country settings,” said the Gates Foundation’s Dan Wattendorf, Director of Innovative Technology Solutions, Global Health. “Handheld, low-cost tools can bring testing out of a laboratory and to the point of need. Digitally enabled tools can help provide objective guidance for those administering a test, reducing procedural errors and facilitating collection of more complete diagnostic information.”
The challenge will address three blood disorders that exact an enormous toll on populations around the globe. Sickle cell disease is a group of inherited red blood cell disorders arising from a single genetic mutation that can cause severe pain and potentially lead to premature death. The condition affects millions of people worldwide, most often those of African ancestry. About 300,000 infants each year have sickle cell disease, 75% of whom reside in sub-Saharan Africa. Without newborn screening programs and early diagnosis, 50-90% of children with the disease in sub-Saharan Africa die before age 5.
Malaria is caused by a parasite that is spread to people through a bite from an infected mosquito. In 2018, approximately 228 million people contracted malaria, with the vast majority of those being in sub-Saharan Africa. The Gates Foundation is committed to implementing new surveillance tools linked to a digital infrastructure with the goal of eradicating malaria entirely.
Anemia affects roughly a third of the world’s population and occurs when the body makes too few red blood cells, destroys too many red blood cells, or loses blood. The most common causes of anemia include iron and other nutritional deficiencies, hemoglobin abnormalities, and infectious diseases, such as malaria, tuberculosis, HIV and parasitic infections. Young children, pregnant women and all women of childbearing age are particularly prone to the effects of anemia.
The challenge will accept applications through June 2, 2020. Register and submit and application through the challenge website, https://www.nibib.nih.gov/NIH-Technology-Accelerator-Challenge. Applicants are reminded to review the challenge guidelines.
Additional partners include the NIH Office of the Director; the National Institute of Allergy and Infectious Diseases; the National Heart, Lung and Blood Institute; the National Institute of Diabetes and Digestive and Kidney Diseases; and the Fogarty International Center.
Sickle Cell Disease: After Years Of Neglect Some Promise For Sufferers
ProMedica joins growing list of hospitals to add adult sickle cell treatment
For years, Dr. Crawford Strunk has felt the Toledo area had a major gap in care available to sickle-cell patients who age out of pediatric clinics.
But in December, the hematologist’s vision to transition his juvenile patients into adult care after they turn 18 finally came to fruition.
ProMedica Toledo Hospital’s new Adult Sickle Cell Clinic housed inside its Oncology and Infusion Center is now treating adult patients who previously had to drive to Detroit, Columbus, Cincinnati, or Dayton to receive specialized care. It’s the latest addition in a field in which more and more adult-care clinics sprout as medicine has advanced life expectancy.
Dr. Strunk, a pediatric hematologist and oncologist at ProMedica Russell J. Ebeid Children’s Hospital, said patients from the Toledo area were driving long distances to get care needed. Now, they’ve got a place right in their backyard.
“This is a center that has the ability to see both youth adolescents as well as older adults,” said Dr. Strunk, who now treats adults as well. “There’s not enough adult providers, so we felt this was a service needed in our area.”
ProMedica’s new clinic is staffed by Dr. Strunk and Dr. Drew Oostra. The clinic provides “comprehensive treatment including transfusions, pain management, and exemplary care for adults,” according to a news release.
The need for more adult care for people who have the sickle-cell trait, a condition that affects approximately 100,000 Americans and 8 percent of African Americans, has heightened over the last 20 years, according to Annie Womack-Ross, the Ohio Sickle Cell and Health Association’s executive director.
When the sickle-cell trait was first identified in 1910, Ms. Womack-Ross said, life expectancy was too short for adult clinics to be needed — and stayed that way for a long time.
“They were not equipped for people to live longer,” she said. “Patients were dying.”
But as medicine progressed, sickle-cell patients began living longer and the gap of care for when patients enter adulthood arose, Ms. Womack-Ross said.
“When you’re dealing with adult patients, you’re dealing with things that you don’t find at pediatric clinics,” she said. “Once a patient transitions into the adult world, that’s where we lose a lot of patients. But it’s 2020. Sickle cell has been diagnosed for a long time. We have more adult patients and we need more adult clinics.”
In nearby Lima, Mercy Health St. Rita’s Family Medicine has also added adult services. Dr. Robert Zukas, an osteopathic physician, said sickle-cell patients in the area were driving to Dayton to get services now offered in Lima.
“The distance made it difficult for them,” he said.
The national median life expectancy is now 42 to 47 years for individuals with sickle-cell disease, according to the American Society of Hematology.
“What previously was never an adult disease, is now an adult disease,” Dr. Zukas said.
Articles in the medical literature
|The Multifaceted Role of Ischemia/Reperfusion in Sickle Cell Anemia J Clin Invest , 130 (3), 1062-1072 2020 Mar 2PMID: 32118586 DOI: 10.1172/JCI133639 Abstract Sickle cell anemia is a unique disease dominated by hemolytic anemia and vaso-occlusive events. The latter trigger a version of ischemia/reperfusion (I/R) pathobiology that is singular in its origin, cyclicity, complexity, instability, perpetuity, and breadth of clinical consequences. Specific clinical features are probably attributable to local I/R injury (e.g., stroke syndromes) or remote organ injury (e.g., acute chest syndrome) or the systematization of inflammation (e.g., multifocal arteriopathy). Indeed, by fashioning an underlying template of endothelial dysfunction and vulnerability, the robust inflammatory systematization no doubt contributes to all sickle pathology. In this Review, we highlight I/R-targeting therapeutics shown to improve microvascular blood flow in sickle transgenic mice undergoing I/R, and we suggest how such insights might be translated into human therapeutic strategies. Publication types Review Full-text links American Society for Clinical Investigation
|Pregnancy in Sickle Cell Trait: What We Do and Don’t Know Br J Haematol 2020 Feb 17[Online ahead of print]Authors Samuel Wilson 1 2 3 , Patrick Ellsworth 1 2 3 , Nigel S Key 1 3 4 Abstract Sickle cell trait (SCT) is the carrier state for sickle cell disease that results from the HBB rs334 missense mutation (p.Glu6Val) in the β-globin chain of haemoglobin. While not associated with any impact on life expectancy, it has been established that SCT is associated with an increased risk of both venous thromboembolism (and in particular, pulmonary embolism) and chronic kidney disease. It is largely unknown what short- or long-term effect, if any, pregnancy has upon the risk or outcomes of these disorders. In addition, SCT has been linked with various adverse outcomes in pregnancy, ranging from maternal complications such as elevated risk of bacteriuria to potentially life-threatening entities such as pre-eclampsia and prematurity. In these scenarios also, no clear association with SCT has been established. Given the high worldwide prevalence of SCT, further studies addressing these issues are warranted. Keywords: chronic kidney disease; pregnancy; sickle cell trait; venous thromboembolism. © 2020 British Society for Haematology and John Wiley & Sons Ltd. 62 references Publication types Review Grant support UO1HL117659/GF/NIH HHS/United States T32HL007149-42/GF/NIH HHS/United States Full-text links Wiley
|Ketamine In Acute and Chronic Pain Management ReviewIn: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2020 Jan–.2020 Feb 18.Authors Vwaire J. Orhurhu 1 , Jacob S. Roberts 2 , Steven P. Cohen 3 Acute and chronic pain remain important health problems both in the United States and worldwide. With the rise in the prevalence of many chronic degenerative diseases across the globe, the distribution and absolute numbers of persons experiencing acute and chronic pain have continued to increase. As a result, pain management has come to the forefront of the public health community. The manifestation of pain itself typically involves the peripheral and central nervous systems. Pain can classify as nociceptive, neuropathic or nocicplastic in origin. Nociceptive pain, also referred to as physiologic pain, results from activation of primary nociceptive afferents by actual or potential tissue-damaging stimuli. In nociceptive pain, large nerve integrity remains preserved as sensory receptors are stimulated within visceral and somatic structures. In contrast to nociceptive pain, neuropathic pain results for direct injury or disease affecting the somatosensory system and tends to be more disabling than nociceptive pain. Neuropathic pain subdivides into peripheral (e.g. diabetic neuropathy) and central (e.g., spinal cord injury or central poststroke pain), while nociceptive pain subcategorizes into somatic or visceral (e.g., inflammatory bowel disease). Recently, the International Association for the Study of Pain added a third category to pain classification of taxonomy for conditions that do not neatly fit into neuropathic or nocicplastic categories. Nociplastic pain refers to pain that arises from altered nociception despite no clear evidence of actual or threatened tissue damage or evidence a disease or lesion directly affecting the somatosensory system. Conditions considered to be nocicplastic pain include fibromyalgia, complex regional pain syndrome (CRPS) type I, and irritable bowel syndrome. One should keep in mind that there are several pain conditions, such as failed back surgery syndrome, that contain elements of more than one pain category, and can be classified as “mixed” pain states. In addition, conditions that are clearly classified as nociceptive and neuropathic often contain overlapping mechanisms with nocicplastic pain in that they involve abnormal nociceptive processing (e.g. amplified pain signals, expansion of receptive fields, decreased descending modulation). Pain can also categorize as acute, chronic, or a combination of these types (e.g. sickle cell crisis). Acute pain arises from a specific disease or injury and its duration is typically self-limited. Acute pain is considered to serve a protective biological purpose and is often associated with muscle spasm and sympathetic nervous system activation. In contrast, chronic pain may be considered a disease state, with its duration outlasting the normal time of healing when associated with disease or injury. Chronic pain may also stem from psychological states and does not serve an apparent biological purpose. Unlike the self-limited nature of acute pain, chronic pain often does not have a recognizable endpoint. Copyright © 2020, StatPearls Publishing LLC. Publication types Review Full-text links StatPearls [Internet]NCBI Bookshelf
|Convergence of Inflammatory Pathways in Allergic Asthma and Sickle Cell Disease Front Immunol , 10, 3058 2020 Jan 24 eCollection 2019Authors Amali E Samarasinghe 1 2 3 , Jason W Rosch 4 Abstract The underlying pathologies of sickle cell disease and asthma share many characteristics in terms of respiratory inflammation. The principal mechanisms of pulmonary inflammation are largely distinct, but activation of common pathways downstream of the initial inflammatory triggers may lead to exacerbation of both disease states. The altered inflammatory landscape of these respiratory pathologies can differentially impact respiratory pathogen susceptibility in patients with sickle cell disease and asthma. How these two distinct diseases behave in a comorbid setting can further exacerbate pulmonary complications associated with both disease states and impact susceptibility to respiratory infection. This review will provide a concise overview of how asthma distinctly affects individuals with sickle cell disease and how pulmonary physiology and inflammation are impacted during comorbidity. Keywords: acute chest syndrome (ACS); asthma; pulmonary inflammation; respiratory infection; sickle cell disease (SCD). Copyright © 2020 Samarasinghe and Rosch. 99 references 2 figures Publication types Review Full-text links Frontiers Media SA Free PMC article
|Curative Options for Sickle Cell Disease: Haploidentical Stem Cell Transplantation or Gene Therapy? Br J Haematol 2020 Feb 7[Online ahead of print]Authors Alexis Leonard 1 2 3 , John Tisdale 1 , Allistair Abraham 3 Abstract Haematopoietic stem cell transplantation (HSCT) is curative in sickle cell disease (SCD); however, the lack of available matched donors makes this therapy out of reach for the majority of patients with SCD. Alternative donor sources such as haploidentical HSCT expand the donor pool to nearly all patients with SCD, with recent data showing high overall survival, limited toxicities, and effective reduction in acute and chronic graft-versus-host disease (GVHD). Simultaneously, multiple gene therapy strategies are entering clinical trials with preliminary data showing their success, theoretically offering all patients yet another curative strategy without the morbidity and mortality of GVHD. As improvements are made for alternative donors in the allogeneic setting and as data emerge from gene therapy trials, the optimal curative strategy for any individual patient with SCD will be determined by many critical factors including efficacy, transplant morbidity and mortality, safety, patient disease status and preference, cost and applicability. Haploidentical may be the preferred choice now based mostly on availability of data; however, gene therapy is closing the gap and may ultimately prove to be the better option. Progress in both strategies, however, makes cure more attainable for the individual with SCD. Keywords: alternative donor; gene therapy; haploidentical; hematopoietic stem cell transplantation; sickle cell disease. © 2020 British Society for Haematology and John Wiley & Sons Ltd. 103 references Publication types Review Grant support American Society of Hematology-Robert Wood Johnson Foundation Full-text links Wiley
|Voxelotor: First Approval Drugs , 80 (2), 209-215 Feb 2020Author Hannah A Blair 1 Affiliation 1 Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. firstname.lastname@example.org.PMID: 32020554 DOI: 10.1007/s40265-020-01262-7 Abstract Voxelotor (Oxbryta™) is a haemoglobin S polymerization inhibitor that has been developed for the treatment of sickle cell disease. In November 2019, voxelotor received its first global approval in the USA for the treatment of sickle cell disease in adults and paediatric patients aged ≥ 12 years. The drug was granted accelerated approval based on the results of the phase III HOPE trial. Phase III clinical development of voxelotor for sickle cell disease is ongoing worldwide. Voxelotor also has Orphan Drug designation and Priority Medicine status in Europe for the treatment of sickle cell disease. This article summarizes the milestones in the development of voxelotor leading to this first approval as a disease-modifying agent for sickle cell disease. 10 references Publication types Review Full-text links Springer
|Cerebral Revascularization for Moyamoya Syndrome Associated With Sickle Cell Disease: A Systematic Review of the Literature on the Role of Extracranial-Intracranial Bypass in Treating Neurologic Manifestations of Pediatric Patients With Sickle Cell Disease World Neurosurg , 137, 62-70 2020 Jan 31[Online ahead of print]Authors Danielle Terrell 1 , Amey R Savardekar 1 , Stephen Garrett Whipple 1 , Rimal H Dossani 1 , Robert F Spetzler 2 , Hai Sun 3 Abstract Moyamoya syndrome (MMS) in patients with sickle cell disease (SCD) accentuates the risk of recurrent strokes. Chronic transfusion therapy (CTT) is an excellent option for preventing recurrent strokes in most patients with SCD. In SCD with MMS, CTT may fail as a long-term solution. Cerebral revascularization, in the form of extracranial-intracranial bypass, has been shown to prevent recurrent strokes in this cohort. We review the evolution of this paradigm shift in the management of SCD-associated MMS. A systematic review, adhering to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses protocol, was conducted. Our primary objectives were 1) to study the evolution of cerebral revascularization techniques in management of MMS in SCD and 2) to analyze the impact of neurosurgical intervention in this high-risk population. Four patients with SCD-associated MMS, who underwent indirect cerebral revascularization at our institute were retrospectively reviewed. A summary of 13 articles chronicling the advent and subsequent evolution of cerebral revascularization as a viable treatment strategy for stroke prevention in SCD-associated MMS is presented. The literature review suggests that early detection and surgical intervention (in addition to CTT) could significantly reduce stroke recurrence and improve neurocognitive outcome. Our short series of 4 patients also had a good outcome and no recurrence of strokes postoperatively. The literature emphasizes the use of a traditional standardized protocol for early identification (transcranial Dopplers, selective magnetic resonance angiography, and CTT). Early treatment and screening that involves early magnetic resonance angiography and referral to neurosurgery for revascularization may be considered for this high-risk population. Keywords: Cerebral ischemic events; Cerebral revascularization; EC-IC bypass surgery; Moyamoya syndrome; Sickle cell disease; Stroke; Transient ischemic attack. Copyright © 2020 Elsevier Inc. All rights reserved. Publication types Review Full-text links Elsevier Science
|Sickle Cell Disease: A Primary Care Update Cleve Clin J Med , 87 (1), 19-27 Jan 2020Authors Grace Onimoe 1 , Seth Rotz 2 Abstract Sickle cell disease (SCD) is the most common hemoglobinopathy in the United States and causes significant disease-related morbidity including multiorgan damage, chronic anemia, and debilitating pain crises. Primary care physicians play a key role in the medical home model of care for adults with SCD. This review focuses on current recommendations for health maintenance and provides a brief summary of disease complications and current updates. Copyright © 2020 The Cleveland Clinic Foundation. All Rights Reserved. Publication types Review Full-text links HighWire
|Sickle Cell Nephropathy ReviewIn: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2020 Jan–.2020 Jan 28.Authors Narothama R. Aeddula 1 , Mainak Bardhan 2 , Krishna M. Baradhi 3 Excerpt Sickle cell disease (SCD), first discovered in West Africa is an autosomal recessive hemoglobin disorder, predominantly affecting persons of African, Mediterranean, Indian, and Middle Eastern descent. It results from the replacement of glutamate for valine at the sixth amino acid of the beta-globin chain. The mutation results in hemoglobin S (HbS) tetramers that accumulate during tissue hypoxia, oxidative stress or dehydration. The accumulation leads to red blood cell sickling, early destruction of erythrocytes, and widespread vaso-occlusive episodes (VOC), subsequently resulting in multiorgan damage. Some of the renal complications, collectively known as sickle cell nephropathy (SCN), include hematuria, hyposthenuria, renal papillary necrosis, proteinuria, renal tubular disorders, acute and chronic kidney injury, sickle cell glomerulopathy, and renal medullary carcinoma. Clinically significant renal involvement occurs more frequently in sickle cell disease than in sickle cell trait or in combined hemoglobinopathies, except renal medullary carcinoma, which appears to be more common among sickle cell trait patients. Natural history of SCD is highly variable with reduced life expectancy with multiorgan damage in symptomatic patients. In general, all patients have a reduced lifespan. Median survival in the United States and Jamaica is 45 to 55 years. Natural history by age in SCD is as follows: The primary cause of death in younger patients is usually infection; whereas, in older patients, the primary cause of death is mostly irreversible organ damage. Copyright © 2020, StatPearls Publishing LLC. Publication types Review Full-text links StatPearls [Internet]NCBI Bookshelf
|Splenic Sequestration Crisis ReviewIn: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2020 Jan–.2020 Jan 19.Authors Ian Kane 1 , Shivaraj Nagalli 2 Excerpt Splenic sequestration is a feared complication of sickle cell anemia that primarily affects young children. It is an acute drop in hemoglobin of 2 g/dL accompanied by splenomegaly. The spleen is at particular risk for complications from sickle cell anemia due to its role as a filter of the blood. The spleen is composed of three areas; white pulp, red pulp, and a transitional zone, and each plays a role within the immune system. A minority of blood flow entering the spleen (approximately 10%) is directed outside of the vessels into the parenchyma of the red pulp, where the red blood cells (RBCs) become exposed to reticuloendothelial cells such as macrophages which clear away abnormal cells or other pathogens. To re-enter the vascular system, the RBCs must squeeze through narrow slits in the endothelium of the venous sinuses. The organization of the white pulp of the spleen is predominantly T-cell periarteriolar sheaths and follicles, consisting of B cells. These B cells are critical in the production of IgM antibodies capable of opsonizing encapsulated bacteria such as Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenza type B. Among children with sickle cell anemia, the spleen gradually loses function and size over the first five years of life due to repeated episodes of autoinfarction and scarring. Children with less severe variants of sickle cell disease such as hemoglobin SC disease or sickle cell-beta thalassemia may have preserved some splenic function into adulthood, and studies have shown that treatment with hydroxyurea can preserve and even result in some recovery of splenic function. Copyright © 2020, StatPearls Publishing LLC. Publication types Review Full-text links StatPearls [Internet]NCBI Bookshelf
|Blood Transfusions for Treating Acute Chest Syndrome in People With Sickle Cell Disease Cochrane Database Syst Rev , 1 (1), CD007843 2020 Jan 16Authors Roya Dolatkhah 1 , Saeed Dastgiri 2 Background: Sickle cell disease is an inherited autosomal recessive blood condition and is one of the most prevalent genetic blood diseases worldwide. Acute chest syndrome is a frequent complication of sickle cell disease, as well as a major cause of morbidity and the greatest single cause of mortality in children with sickle cell disease. Standard treatment may include intravenous hydration, oxygen as treatment for hypoxia, antibiotics to treat the infectious cause and blood transfusions may be given. This is an update of a Cochrane Review first published in 2010 and updated in 2016. Objectives: To assess the effectiveness of blood transfusions, simple and exchange, for treating acute chest syndrome by comparing improvement in symptoms and clinical outcomes against standard care. Search methods: We searched The Cochrane Cystic Fibrosis and Genetic Disorders Group’s Haemoglobinopathies Trials Register, which comprises references identified from comprehensive electronic database searches and handsearching of relevant journals and abstract books of conference proceedings. Date of the most recent search: 30 May 2019. Selection criteria: Randomised controlled trials and quasi-randomised controlled trials comparing either simple or exchange transfusion versus standard care (no transfusion) in people with sickle cell disease suffering from acute chest syndrome. Data collection and analysis: Both authors independently selected trials and assessed the risk of bias, no data could be extracted. Main results: One trial was eligible for inclusion in the review. While in the multicentre trial 237 people were enrolled (169 SCC, 42 SC, 15 Sβ⁰-thalassaemia, 11Sβ+-thalassaemia); the majority were recruited to an observational arm and only ten participants met the inclusion criteria for randomisation. Of these, four were randomised to the transfusion arm and received a single transfusion of 7 to 13 mL/kg packed red blood cells, and six were randomised to standard care. None of the four participants who received packed red blood cells developed acute chest syndrome, while 33% (two participants) developed acute chest syndrome in standard care arm. No data for any pre-defined outcomes were available. Authors’ conclusions: We found only one very small randomised controlled trial; this is not enough to make any reliable conclusion to support the use of blood transfusion. Whilst there appears to be some indication that chronic blood transfusion may play a roll in reducing the incidence of acute chest syndrome in people with sickle cell disease and albeit offering transfusions may be a widely accepted clinical practice, there is currently no reliable evidence to support or refute the perceived benefits of these as treatment options; very limited information about any of the potential harms associated with these interventions or indeed guidance that can be used to aid clinical decision making. Clinicians should therefore base any treatment decisions on a combination of; their clinical experience, individual circumstances and the unique characteristics and preferences of adequately informed people with sickle cell disease who are suffering with acute chest syndrome. This review highlights the need of further high quality research to provide reliable evidence for the effectiveness of these interventions for the relief of the symptoms of acute chest syndrome in people with sickle cell disease. Copyright © 2020 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd. Conflict of interest statement There are no financial conflicts of interest and the review authors declare that they do not have any associations with any parties who may have vested interests in the results of this review.Update of Blood Transfusions for Treating Acute Chest Syndrome in People With Sickle Cell Disease S Dastgiri et al. Cochrane Database Syst Rev (8), CD007843. 2016. PMID 27574910. – ReviewWe found only one very small randomised controlled trial; this is not enough to make any reliable conclusion to support the use of blood transfusion. Whilst there appears … Publication types Review Research Support, Non-U.S. Gov’t Full-text links Wiley
|The Evolving Pharmacotherapeutic Landscape for the Treatment of Sickle Cell Disease Mediterr J Hematol Infect Dis , 12 (1), e2020010 2020 Jan 1 eCollection 2020Author Samir K Ballas 1 Affiliation 1 Cardeza Foundation for Hematologic Research, Department of Medicine, Sidney Kimmel Medical College, Thomas Jefferson University, Philadelphia, PA, USA.PMID: 31934320 PMCID: PMC6951351 DOI: 10.4084/MJHID.2020.010 Abstract Sickle cell disease (SCD) is an extremely heterogeneous disease that has been associated with global morbidity and early mortality. More effective and inexpensive therapies are needed. During the last five years, the landscape of the pharmacotherapy of SCD has changed dramatically. Currently, 54 drugs have been used or under consideration to use for the treatment of SCD. These fall into 3 categories: the first category includes the four drugs (Hydroxyurea, L-Glutamine, Crizanlizumab tmca and Voxelotor) that have been approved by the United States Food and Drug Administration (FDA) based on successful clinical trials. The second category includes 22 drugs that failed, discontinued or terminated for now and the third category includes 28 drugs that are actively being considered for the treatment of SCD. Crizanlizumab and Voxelotor are included in the first and third categories because they have been used in more than one trial. New therapies targeting multiple pathways in the complex pathophysiology of SCD have been achieved or are under continued investigation. The emerging trend seems to be the use of multimodal drugs (i.e. drugs that have different mechanisms of action) to treat SCD similar to the use of multiple chemotherapeutic agents to treat cancer. Keywords: Pharmacotherapeutic; Sickle cell disease. Conflict of interest statement Competing interests: The authors declare no conflict of Interest.202 references 3 figures Publication types Review Full-text links Catholic University in Rome, Institute of Hematology Free PMC article
|Innate Immune Cells, Major Protagonists of Sickle Cell Disease Pathophysiology Haematologica , 105 (2), 273-283 2020 Jan 31Authors Slimane Allali 1 2 3 , Thiago Trovati Maciel 2 3 , Olivier Hermine 2 3 4 , Mariane de Montalembert 1 3 Abstract Sickle cell disease (SCD), considered the most common monogenic disease worldwide, is a severe hemoglobin disorder. Although the genetic and molecular bases have long been characterized, the pathophysiology remains incompletely elucidated and therapeutic options are limited. It has been increasingly suggested that innate immune cells, including monocytes, neutrophils, invariant natural killer T cells, platelets and mast cells, have a role in promoting inflammation, adhesion and pain in SCD. Here we provide a thorough review of the involvement of these novel, major protagonists in SCD pathophysiology, highlighting recent evidence for innovative therapeutic perspectives. Copyright© 2020 Ferrata Storti Foundation. 100 references 4 figures Publication types Review Full-text links HighWire Free PMC article
|Acute Chest Syndrome ReviewIn: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2020 Jan–.2020 Jan 5.Authors Amanda Friend 1 , Daniel Girzadas 1 Excerpt Acute chest syndrome (ACS) is the result of various inciting events causing vaso-occlusion within the pulmonary vasculature of patients with sickle cell disease (SCD). ACS can occur in any SCD phenotype. However, it is most common in HbSS. ACS can progress quickly and is the most common cause of death in patients with SCD. Therefore, it is important to make the diagnosis and begin treatment as soon as possible. Copyright © 2020, StatPearls Publishing LLC. Publication types Review Full-text links StatPearls [Internet]NCBI Bookshelf
Sickle Cell Conferences and Events
CME/CEU Symposium on Sickle Cell Disease March 27, 2020 8:30 – 4:30 at Brookdale University Hospital & Medical Center One Brookdale Plaza Brooklyn, New York 11212 For Information Call: (718) 240-5904 or (718) 613-6874
The Hemoglobinopathy Counselor Training Course will be held in Cincinnati on April 15-16, 2020. The two-day course, presented by the Cincinnati Comprehensive Sickle Cell Center, will be held at Cincinnati Children’s Hospital Medical Center. The course registration fee is $250. The deadline to register is April 1, 2020 and registration space is limited. For more information, please email: SCDEvents@cchmc.org. Registration is available online at www.cincinnatichildrens.org/HemoglobinopathyCTC.
4th Global Congress on Sickle Cell Disease – Call for Abstracts
Abstract submissions are now open for the Global Sickle Cell Disease Network’s 4th Global Congress on Sickle Cell Disease (SCD), which will take place April 7-9, 2020 in Paris, France. The focus of the meeting will be on the compelling need to find one united and coordinated voice for SCD worldwide. Abstract submissions for the Congress will close February 10, 2020. Decision notifications will be sent out on March 9, 2020.The two best abstracts submitted by young investigators (PhD students or post doctorate trainees) will be selected for Young Investigator Prize presentations in the plenary session of the congress.
For more information, and to submit an abstract click here.
First IASCNAPA Conference
The International Association of Sickle Cell Nurses and Professional Associates (IASCNAPA) is hosting a Sickle Cell Disease Conference: Treating the Whole Person, April 17, 2020in Memphis, TN. This is an all-day conference designed to provide information on treating the whole person with sickle cell disease. Experts will provide practical tools using case studies and interactive exercises. Sessions will include pain management and mental health tools; transition skills; and “out of the box” techniques such as virtual reality, and drama therapy. Practical sickle cell resources for nurses, advanced practice practitioners and other healthcare professionals will also be provided. Click hereto learn more and to register. www.iascnapa.org
Symposium on heart complications in SCD on May 22ndat St. Jude in Memphis TN. Registration is free. For information: https://stjude.cloud-cme.com/default.aspx?P=5&EID=3484
Foundation for Sickle Cell Disease Research Symposium
The Foundation for Sickle Cell Disease Research (FSCDR) is committed to supporting innovative research in sickle cell disease (SCD) to help maximize quality of life and improve survival for the generations of people affected by SCD. The Symposium focuses on interactive education, sharing of best practices, and exploring novel approaches to dealing with SCD that goes beyond theory to develop practical, real-life solutions. The next annual FSCDR symposium will take place June 12-14, 2020, in Fort Lauderdale, FL. Learn more and register today. https://fscdr.org/the-symposium/