Swedish Study Identifies MIS Risk Factors Among Pediatric Patients
Multisystem inflammatory syndrome (MIS) in children is a rare condition known to be associated with COVID-19, but more research is needed to identify at-risk patients and guide public health interventions.
Population-Based Study Finds Racial Disparities in MDS Outcomes
Research presented at the 2022 American Society of Clinical Oncology Annual Meeting shows White patients with myelodysplastic syndrome (MDS) have worse overall survival outcomes than African American patients, contrary to previous findings in other cancer types.
FDA Offers Plan for Developing Drugs for Neurodegenerative Disease, Including ALS
The FDA released an action plan to address the Accelerating Access to Critical Therapies for ALS (amyotrophic lateral sclerosis) Act.
Poor Health-Related Quality of Life, Many Burdens in Rare Diseases, Study Finds
People with rare diseases experience significant diagnostic delays, high out-of-pocket costs, travel burden for specialty care, and insufficient dental and psychological support, according to new findings.
Transfusion-Dependent Patients With MDS Vary in Treatment Experiences, Preferences
Transfusion dependence is common for patients with myelodysplastic syndromes (MDS), but real-world data show inconsistent patient experiences and varied transfusion practices between countries.
Autoimmunity May Spark Thrombosis in Patients With Polycythemia Vera, Study Says
Physicians should consider measuring anti-endothelial cell antibodies in patients with polycythemia vera, the investigators suggested.
Vitamin D May Affect GVHD Rates in Stem Cell Transplantation, Review Finds
Study data are mixed, but some evidence suggests the administration of vitamin D may work as a low-risk prophylactic against graft-versus-host disease (GVHD).
Study: Suggested Cyclosporine Levels Posttransplant May Be Too Low to Prevent GVHD
Preventing graft-versus-host disease (GVHD) after allogeneic stem cell transplant is a key aspect of patient management, but current cyclosporine concentration guidelines may not be sufficient, according to a recent study.
Individualized Antimicrobial Treatment, Prophylactic Regimens May Help With GVHD After Liver Transplant
A recent study aimed to provide insight and guidance for managing graft-versus-host disease in patients after liver transplantation.
Study Finds Link Between Particulate Matter Composition, ALS Aggravation
A recent study found positive associations between the organic matter component of particulate matter and disease aggravation in amyotrophic lateral sclerosis (ALS).
Study Combines Neurological Assessments to Better Identify SMA in Newborns
A small study aimed to increase the likelihood of detecting minor neurological symptoms of spinal muscular atrophy via a combination of tests specifically suited to newborns.
Study Suggests Ocular GVHD Is Underdiagnosed in Patients After Hematopoietic Stem Cell Transplantation
A survey of blood and bone marrow transplant patients found that many who fit current criteria for ocular graft-versus-host disease have not been diagnosed.
CLAD, Pulmonary GVHD Have Similar Clinical Traits, Research Needs
Fine-tuning clinical phenotypes and identifying effective treatments remain challenging in chronic lung allograft dysfunction (CLAD) after lung transplantation and chronic graft-versus-host disease (GVHD) after allogeneic hematopoietic stem-cell transplantation.
RAS Inhibitors May Hold Potential as Preventive for Acute Chest Syndrome in Sickle Cell Disease
A retrospective study assessed hospital readmission rates in patients with sickle cell disease and acute chest syndrome who took angiotensin-converting enzyme inhibitors or angiotensin II receptor blockers vs those who did not.
New FDA Program to Focus on Driving Rare Disease Drug Development
In an effort to bolster development of treatments for rare diseases, the FDA announced the creation of the new Accelerating Rare disease Cures (ARC) program.
Review Highlights Treatment Landscape, Insights on Pregnancy Management for ET or PV
Polycythemia vera and essential thrombocythemia are both slow-progressing conditions and treated mainly with symptom management, but novel agents and gene targets have changed the landscape in recent years.
Poorer Outcomes Associated With Recurrent aGVHD Warrant Independent Recognition, Says Study
After a median follow-up of 535 days, analysis showed that recurrent acute graft-versus-host disease (RaGVHD) carried several implications for survival, leading the researchers to call for further risk stratification of aGVHD.
Real-world Study Shows Ruxolitinib May Reduce Arterial Thrombosis in Polycythemia Vera
Previous studies have shown ruxolitinib effective in symptom relief and hematocrit control for hydroxyurea-resistant polycythemia vera, but its role in disease progression is not yet clear.
MoCA Scores in HD Correlate With Caudate Atrophy, Cortical Thinning, Says New Research
The findings suggest that the tool can be used to not only assess cognitive impairment in Huntington disease (HD) but also to detect brain atrophy patterns associated with cognitive status in these patients.
Decreased Levels of Zinc Associated With Worse SCD Severity in Children, Adolescents
A review of 41 articles indicated that leveraging zinc supplementation in patients with sickle cell disease (SCD) is a safe and potentially effective approach for these patients.
Study Demonstrates Potential of Composite End Point in Assessing Treatment for PNH
A recent study found that a composite end point developed by the researchers gave a single and simultaneous measurement of the overall benefit of treatment for paroxysmal nocturnal hemoglobinuria (PNH).
ICER Examines Possible Policies Aimed at Sustaining Future Rare Disease Drug Development
The Institute for Clinical and Economic Review (ICER) and researchers at NORC at the University of Chicago released a white paper that looks at the future of affordability and sustainability of drug development for rare disease through the lens of 4 policy proposals.
Researchers Identify Protein That May Be Indicator of Treatment Response in SMA
With responses to current treatments varying among patients with spinal muscular atrophy (SMA), the researchers emphasized the importance of more prognostic markers for improved decision making.
Study Adds to Limited Data on SMA Genetic Landscape in Africa
Acknowledging the small number of genetic reports used in their study, the researchers attribute the scarce amount of data to a lack of resources and limited access to genetic screening across Africa.
Case Report and Review Highlight Importance of Identifying Osteolytic Lesions in MPNs
A recent report details the first reported case of an osteolytic lesion in polycythemia vera and reviews current literature on osteolytic lesions in myeloproliferative neoplasms (MPNs) overall.
What We’re Reading: Epilepsy Drug Approval; Free COVID-19 Testing Ends; EPA Sued
The FDA approved a drug to treat a rare form of childhood epilepsy; uninsured Americans will no longer have access to free COVID-19 tests; a conservation group is suing the Environmental Protection Agency (EPA) over failure to protect rivers from pollution.
What We’re Reading: FDA Reviews ALS Drug; Vaccines Lower Long COVID-19; New COTA President, CEO
The FDA accepts an application for a drug to treat amyotriphic lateral sclerosis (ALS) for review; vaccines have been found to reduce people’s risk for developing long COVID-19; COTA announces Miruna Sasu, PhD, as its new president and CEO.
Case Series Assesses Clinical Features of Pediatric APS
To enable a more personalized and proactive approach to pediatric antiphospholipid syndrome (APS), investigators assessed electronic medical records of 21 children.
Investigators Propose New Tool for Establishing Gene-Disease Relationships in Rare Diseases
Advances in whole genome sequencing are helpful in linking genetic variants with diseases, but the process is more challenging in cases of rare and ultra-rare diseases.
Potential Link Found Between Thrombotic Events, Mortality in Patients With PV, ET
Patients with polycythemia vera (PV) or essential thrombocythemia (ET) had a higher risk of thrombotic events than the general population, which was associated with mortality in a recent study.
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