Sickle Cell Anemia (Hemoglobin S Disease)

A disease characterized by chronic hemolytic anemia, episodic painful crises, and pathologic involvement of many organs. It is the clinical expression of homozygosity for hemoglobin S.

Also Known As:

Hemoglobin S Disease; Anemia, Sickle Cell; HbS Disease; Sickle Cell Disease; Sickle Cell Disorders; Sickling Disorder Due to Hemoglobin S; Anemias, Sickle Cell; Cell Disease, Sickle; Cell Diseases, Sickle; Cell Disorder, Sickle; Cell Disorders, Sickle; Disease, Hemoglobin S; Hemoglobin S Diseases; Sickle Cell Anemias; Sickle Cell Diseases; Sickle Cell Disorder

Networked: 9866 relevant articles (486 outcomes, 1157 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1.	Thalassemia
2.	Pain (Aches)
3.	Stroke (Strokes)
4.	beta-Thalassemia (Cooley's Anemia)
5.	Infections

Experts

1.	Ware, Russell E: 91 articles (01/2022 - 01/2002)
2.	Gladwin, Mark T: 88 articles (01/2022 - 02/2002)
3.	Kato, Gregory J: 75 articles (01/2022 - 01/2005)
4.	Steinberg, Martin H: 74 articles (01/2022 - 05/2002)
5.	Ballas, Samir K: 74 articles (07/2021 - 02/2002)
6.	Gordeuk, Victor R: 70 articles (01/2022 - 01/2008)
7.	DeBaun, Michael R: 69 articles (05/2022 - 10/2004)
8.	Hankins, Jane S: 59 articles (07/2022 - 10/2005)
9.	Tisdale, John F: 59 articles (01/2022 - 02/2002)
10.	Thein, Swee Lay: 55 articles (01/2022 - 03/2005)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Sickle Cell Anemia:

1.	Hydroxyurea (Hydrea)FDA LinkGeneric 04/01/2018 - "Significant reduction in morbidity and mortality have been documented in patients with sickle cell disease (HbSS) by most of the studies using hydroxyurea at a dose of 25-35 mg/kg/day or maximum tolerated dose. " 12/01/2018 - "Although hydroxyurea is proven effective in treatment of sickle cell anemia (SCA) and is widely prescribed in high-income countries, due to questions about feasibility of treating large numbers of patients in resource-limited health systems, its use is limited in sub-Saharan Africa (SSA), where most children with SCA live. " 04/01/2013 - "Hydroxyurea (HU) is highly effective treatment for sickle cell disease (SCD). " 07/01/1997 - "Children with sickle cell anemia provide the best opportunity to assess the efficacy of hydroxyurea (HU) in preventing complications and progressive organ damage. " 01/01/2020 - "Hydroxyurea has proven safety, feasibility, and efficacy in children with sickle cell anemia in sub-Saharan Africa, with studies showing a reduced incidence of vaso-occlusive events and reduced mortality. "
2.	Fetal Hemoglobin (Hemoglobin F)IBA 01/01/2014 - "Decades of research have established that the most effective treatment for sickle cell disease (SCD) is increased fetal hemoglobin (HbF). " 02/01/2008 - "Increasing hemoglobin F (HbF) appears to be beneficial for patients with sickle cell anemia. " 10/01/2004 - "Increased levels of fetal hemoglobin (HbF) are clinically beneficial in patients with sickle cell disease. " 03/15/1999 - "High levels of fetal hemoglobin (Hb F) protect from many of the complications of sickle cell disease and lead to improved survival. " 08/31/2023 - "Infusion of autologous OTQ923 into three participants with severe sickle cell disease resulted in sustained induction of red-cell fetal hemoglobin and clinical improvement in disease severity. "
3.	Hemoglobins (Hemoglobin)IBA 01/01/2021 - "Gene therapy as a potential cure for sickle cell disease (SCD) has long been pursued, given that this hemoglobin (Hb) disorder results from a single point mutation. " 01/01/2023 - "We computed PRS in African ancestry populations using existing European-only versus ancestrally diverse genetic studies; the increased diversity produced the largest accuracy gains for hemoglobin concentration and white blood cell count, reflecting large-effect ancestry-enriched variants in genes known to influence sickle cell anemia and the allergic response, respectively. " 01/01/2022 - "The study consisted of HemoType SC rapid test screening for sickle cell disease and then diagnostic confirmation by hemoglobin electrophoresis. " 01/01/2021 - "Along with the high occurrence of sickle cell disorders in the study area, some other rare hemoglobin disorders are also prevalent which calls for a large community-based cohort study." 06/12/2020 - "In this work we provide preclinical data to support initiation of a first-in-human trial for sickle cell disease (SCD) using an approach that relies on reversal of the developmental fetal-to-adult hemoglobin switch. "
4.	GlobinsIBA 01/01/2012 - "Activation of γ-globin gene expression in adults is known to be therapeutic for sickle cell disease. " 06/14/2019 - "Clinical trials are currently underway to treat sickle cell disease using LVs expressing designed anti-sickling globin genes. " 05/01/2019 - "The aim of this study was to determine the prevalence of sickle cell anemia and β-globin haplotype distribution in newborns from the Bengo region. " 01/01/2016 - "Furthermore, this study shows that the sickle Hb [HbS, occurs due to single nucleotide polymorphism at A>T of β-globin gene of Hb and causes sickle cell disease (SCD)] also bound to GP1bα and activated platelets in a concentration-dependent manner. " 07/01/2014 - "A double-blind, placebo-controlled phase II study of the efficacy and safety of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease."
5.	Sickle HemoglobinIBA 01/01/1992 - "Since cell shrinkage adversely influences red cell rheology, it is unlikely that this class of compounds, despite its ability to inhibit hemoglobin S polymerization, will prove useful in the treatment of sickle cell disease." 09/01/2018 - "Pre- and post-transfusion hemoglobin S (HbS) levels are used to document the efficacy of red blood cell exchange (RCE) in patients with sickle cell disease (SCD). " 08/01/2008 - "Using this improved approach, we also generated hES-like cells from homozygous fibroblasts containing the sickle cell anemia mutation Hemoglobin Sickle. " 10/01/2002 - "We have shown that decreased baseline oxygen saturation in sickle cell disease is related to abnormal hemoglobin S levels, and oxygen saturation can be improved with erythrocytapheresis, independent of any change in the total hematocrit." 02/14/2023 - "Despite a wealth of exploratory plasma metabolomics studies in sickle cell disease (SCD), no study to date has evaluate a large and well phenotyped cohort to compare the primary erythrocyte metabolome of hemoglobin SS, SC and transfused AA red blood cells (RBCs) in vivo . "
6.	Penicillins (Penicillin)FDA Link 02/01/1999 - "Since the introduction of penicillin prophylaxis policies, newborn screening, new immunizations, and comprehensive medical care centers, the survival of young children with sickle cell disease has improved. " 06/01/2013 - "Since the publication of the Prophlyactic Penicillin Study II in 1995, the management of penicillin prophylaxis for children with sickle cell disease (SCD) after 5 years of age has been controversial. " 01/01/2017 - "Once a fatal disease of childhood, more than 95% of patients born today with sickle cell disease (SCD) in developed countries are expected to survive into adulthood, largely because of improvements in supportive and preventive care (newborn screening, penicillin prophylaxis, transcranial Doppler (TCD) screening). " 06/01/2013 - "Prophylactic penicillin after 5 years of age in patients with sickle cell disease: a survey of sickle cell disease experts." 01/13/2012 - "Nasopharyngeal carriage of penicillin resistant pneumococci in Ugandan children with homozygous sickle cell disease is high. "
7.	ZincIBA 01/01/1989 - "Plasma zinc concentrations were determined in patients with sickle cell anemia and in disease free controls in the Eastern province of Saudi Arabia. " 10/01/1988 - "We examined the zinc status of 80 children with sickle cell disease (SCD) and 44 disease-free sibling controls aged 3 to 18 years. " 06/28/2013 - "The authors recommend that more trials on zinc supplementation in thalassaemia and sickle cell disease be conducted given that the literature has shown the benefits of zinc in these types of diseases." 06/28/2013 - "Randomised, placebo-controlled trials of zinc supplements for treating thalassaemia or sickle cell disease administered at least once a week for at least a month. " 12/01/2002 - "This study was designed to explore the use of plasma zinc in determining zinc deficiency in children with sickle cell disease-SS (SCD-SS) as indicated by a growth response to zinc supplementation. "
8.	IronIBA 07/14/2023 - "In the last few decades, the life expectancy of patients with transfusion-dependent thalassaemia (TDT) and sickle cell disease (SCD) has improved significantly, in part because of improved iron chelation. " 10/01/2023 - "The included studies also described the influence of sickle cell anemia on serum iron [SMD = 1.52; (95% CI: 1.32-1.76); (p < 0.01)] compared to normal subjects. " 01/01/2020 - "The aim of our study was to assess the relationships between pancreatic T2* values and pancreatic iron loading with cardiac dysfunctions and liver and cardiac iron among patients with β-thalassaemia major (βTM) and sickle cell disease (SCD). " 01/01/2020 - "The aim of our study was to investigate cardiac function and cardiac iron and their relation to pancreatic iron among patients with β-thalassaemia major (βTM) and sickle cell disease (SCD). " 01/01/2020 - "Study of Iron Status in Sickle cell disorder."
9.	voxelotorIBA 01/01/2021 - "Voxelotor: A Novel Treatment for Sickle Cell Disease." 06/01/2020 - "Food and Drug Administration approved voxelotor (Oxbryta) as a novel treatment for sickle cell disease. " 01/01/2022 - "Safety and efficacy of voxelotor in pediatric patients with sickle cell disease aged 4 to 11 years." 01/01/2023 - "Patient-Reported Experiences in Voxelotor-Treated Children and Adults with Sickle Cell Disease: A Semistructured Interview Study." 05/01/2021 - "In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin polymerisation inhibitor, was approved in the USA for the treatment of sickle cell disease in patients aged 12 years and older on the basis of HOPE trial data. "
10.	Oxygen (Dioxygen)IBA 01/01/2021 - "It should also be helpful in analyzing oxygen binding and in vivo delivery in future investigations of oxygen-affinity-modifying drugs for sickle cell disease." 01/01/2021 - "This study tested the hypothesis that elevated cerebral blood flow and oxygen extraction fraction are associated with lower executive function in individuals with sickle cell anemia. " 01/01/2021 - "(4) Conclusions: The present study demonstrates that oxygen gradient ektacytometry parameters are affected by sickle cell disease (SCD) genotype and treatment." 10/07/2010 - "We have developed a microfluidic approach to study the sickling of red blood cells associated with sickle cell anemia by rapidly varying the oxygen partial pressure within flowing microdroplets. " 06/15/1993 - "This study provides evidence that Hb oxygen desaturation is not a universal finding among children with sickle cell disease and identifies factors associated with Hb oxygen desaturation. "

Therapies and Procedures

1.	Therapeutics 01/01/2023 - "Because of the unique biology of sickle cell disease (SCD) as well as the societal disadvantages and racial inequities suffered by these patients, individuals with SCD have not benefited from the same remarkable advances in care and therapeutics as those with other hematologic disorders. " 02/01/2009 - "While supportive care remains the best option for most well children with sickle cell disease (SCD), increasing awareness of early signs of chronic organ damage in childhood has focused attention on therapy which modulates the natural history of the disease. " 01/01/2001 - "With increased recognition of the profound morbidity of sickle cell disease and with growing evidence of the efficacy of transfusion therapy in prevention and treatment of sickle cell complications, most patients now receive intermittent transfusion therapy. " 06/12/2012 - "Better treatment of sickle cell disease with transfusion therapy after year 1998 (p = 0.007) and improved diagnostic accuracy of MRI (p = 0.009) may partially explain these trends. " 04/01/2009 - "Although the pathophysiology and molecular basis of sickle cell disease (SCD) were described more than half a century ago, an effective and safe therapy is not yet available. "
2.	Blood Transfusion (Blood Transfusions) 03/16/2013 - "No consensus exists on whether preoperative blood transfusions are beneficial in patients with sickle-cell disease. " 09/01/2023 - "It remains likely that in lower-income countries effort should be concentrated on developing laboratory services and blood transfusion, to allow safe and effective support for the assessment of treatment of anemia, sickle cell disease, maternal and child health and urgent surgery and obstetric services. " 05/26/2001 - "Screening for, and appropriate management of, nocturnal hypoxaemia might be a safe and effective alternative to prophylactic blood transfusion for primary prevention of CNS events in sickle-cell disease." 11/01/2020 - "Blood transfusion is the cornerstone treatment to reduce the clinical severity of sickle cell disease (SCD), but we need to maintain the haematocrit (Hct) within an acceptable range to avoid a deleterious increase in blood viscosity. " 12/01/2013 - "Blood transfusion safety in sickle cell anemia patients in Mali should be improved by the introduction of at least the technique for detecting the viral genome in the panel of screening tests and a policy of transfusions of blood units only from regular blood donors. "
3.	Hematopoietic Stem Cell Transplantation 12/01/2022 - "Although sickle cell disease can be cured using allogeneic hematopoietic stem cell transplantation and possibly gene therapy and gene editing, these treatments remain unavailable to most patients. " 01/01/2019 - "To date, sickle cell disease has been cured by hematopoietic stem cell transplantation in approximately 1,000 people, most of whom were children, and significantly ameliorated by gene therapy in a handful of subjects who have only limited follow-up thus far. " 11/01/2022 - "Allogeneic hematopoietic stem cell transplantation (HSCT) provides a cure for patients with sickle cell disease (SCD). " 08/01/2017 - "Allogeneic hematopoietic stem cell transplantation (HSCT) offers the possibility of cure for sickle cell disease (SCD) patients. " 11/01/2016 - "As the safety and availability of allogeneic hematopoietic stem cell transplantation (HSCT) have improved, this procedure is becoming a viable option for nonmalignant conditions such as sickle cell disease (SCD). "
4.	Cell Transplantation 01/01/2018 - "Hematopoietic cell transplantation (HCT) using an HLA-identical sibling donor offers a very high likelihood of cure with good outcomes for patients with sickle cell disease (SCD), and alternative donor HCT for SCD is an area of active clinical research. " 08/01/2011 - "The lack of matched sibling donors poses a significant barrier to utilizing hematopoietic cell transplantation (HCT), the only proven cure for children with sickle cell disease (SCD). " 12/19/2019 - "Sickle cell disease (SCD) leads to significant morbidity and early mortality, and hematopoietic cell transplantation (HCT) is the only widely available cure, with impacts seen on SCD-related organ dysfunction. " 01/01/2023 - "Hematopoietic cell transplantation (HCT) is an established cure for sickle cell disease (SCD) supported by long-term survival, but long-term organ function data are lacking. " 12/09/2022 - "Allogeneic hematopoietic cell transplantation, gene therapy, and gene editing offer a potential cure for sickle cell disease (SCD). "
5.	Splenectomy 01/01/2006 - "Open splenectomy remains the gold standard for patients with sickle cell disease requiring splenectomy." 11/07/2017 - "However, there is a lack of evidence from trials showing that splenectomy improves survival and decreases morbidity in people with sickle cell disease. " 03/15/2016 - "The aim of this study was to study hospital records for all children aged 2 to 12 year old with Sickle cell disease who underwent splenectomy in Tabuk in Saudi Arabia. " 09/07/2015 - "However, there is a lack of evidence from trials showing that splenectomy improves survival and decreases morbidity in people with sickle cell disease. " 04/01/2015 - "In this study we examined the patients with sickle cell disease and partial splenectomy and compared the clinical and laboratory results with non-splenectomized patients. "