Novel Ruxolitinib Combo Shows Promise in Myelofibrosis
Presented as an abstract at the European Hematology Association 2021 Virtual Congress, the study results showed the novel combination resulted in early disease modification among 34 patients.
Greater Understanding of PV, ET Drives Potential New Treatment Options
The novel agents are a result of a greater understanding of the conditions and may modify the diseases’ course and cytoreduction, researchers said.
European Data Initiative Aims to Solve Rare Diseases
Using the new data initiative, researchers hope to diagnose unsolved cases of rare diseases.
Peer-Delivered Intervention Can Increase Patients’ Acceptance of Rare Diseases
Results of a clinical trial indicate peer-delivered interventions can help patients with rare diseases achieve disease acceptance.
Early Initiation of Ruxolitinib Reduces Risk, Severity of aGVHD
The study of 57 patients with acute graft-versus-host disease (aGVHD) also showed that patients tolerated the treatment, offering promise for preventing and managing the complication of transplantation, which affects up to half of patients.
Novel Approaches Needed to Mitigate Myelofibrosis Persistence, Say Researchers
According to researchers, type 2 inhibitors do not seem to result in persistence of disease. In one mouse model, the inhibitor type was shown to also have a greater effect on disease biology.
Caring for Children With AADC-d Impacts Parental Ability to Work, Study Shows
The study portrays the intense need for daily care experienced by children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) and their families.
Trial Finds Rituximab Is Superior to Mycophenolate Mofetil in Treating Pemphigus Vulgaris
A phase 3 trial explored which treatment option for pemphigus vulgaris, a rare skin disease, resulted in better outcomes after 52 weeks.
With No Treatment for Tangier Disease, Management Focuses on Preventing Atherosclerosis
There are no curative treatments for Tangier disease, but gene therapy for ABCA1 has been highlighted as a potential avenue for treating the disease, say the researchers of the paper.
The Economic Burden and Impact on HRQOL of Sickle Cell Disease
Patients with sickle cell disease face substantial health care utilization and impaired health-related quality of life (HRQOL).
Real-world Evidence Supports Conditional Use of IV Formula in Hospitalized Infants With MSUD
Hospitalized infants with the rare metabolic disorder maple syrup urine disease (MSUD), who are intolerant to oral or enteral administration of branched-chain amino acid-free formula, may benefit from an intravenous formulation.
Complications of Steroid Use in GVHD Associated With Higher Health Care Usage, Costs
For patients with acute and chronic graft-vs-host disease (GVHD), systemic treatment with steroids is the standard of care, and although steroid-related complications are common, the associated health care resource utilization and costs are not well documented.
How Does Imaging Perform in Diagnosing MPNs?
The current standard calls for bone marrow biopsies, which are invasive and costly.
Novel Approaches to Treating Polycythemia Vera Needed, Say Researchers
The current treatment landscape of polycycthemia vera (PV) hinges on treatments like hydroxyurea and ruxolitinib, the latter having emerged as an effective second-line therapy in patients with severe pruritis, symptomatic splenomegaly, or post-PV myelofibrosis symptoms.
Assessing Best Practices in Managing Pregnancy, Myeloproliferative Neoplasms
Researchers discuss the unique fetal and maternal challenges for pregnant women with myeloproliferative neoplasms, with insight and recommendations provided on the potential benefit of aspirin therapy, cytoreductive therapy, and systemic anticoagulation.
Including Indirect Costs, Rare Disease Toll Nearly $1 Trillion, Report Says
According to the report, direct medical costs represented less than half ($418 billion, or 43%) of overall annual costs.
Several Treatment Classes Show Promise in Treatment of PV
The review comes amid growing attention directed at identifying treatments that can prevent progression of polycythemia vera (PV) to myelofibrosis or an aggressive form of acute myeloid leukemia.
What We're Reading: UK Variant Not Linked With Severe Disease; COVID-19 Hospitalization in Children; Therapy Setbacks in Huntington Disease
A new study finds no link between the UK COVID-19 variant and more severe disease; study finds more than 1 in 10 US children with COVID-19 hospitalized; therapy setbacks in Huntington disease.
Ruxolitinib Drives High Rate of Transplantation for Patients With Myelofibrosis
The study also indicated that the type of transplantation donor impacted outcomes following the procedure.
Case of Shulman Syndrome Highlights Importance of MRI in cGVHD Treatment Decisions
According to authors, magnetic resonance imaging (MRI) should be used when incomplete signs of the rare complication are presented to help guide the skin biopsy.
Research Highlights Mast Cell Involvement in cGVHD
According to a recent review, data suggest mast cells have an important role in tissue homeostasis and wound healing, with mass cell dysregulation potentially leading to fibrotic disease.
Report Lauds Orphan Drug Act but Notes Most Therapies Are Blocked From Competition
Although the FDA has approved 599 orphan drugs since 1983, patent laws and other exclusivities keep most from any competition.
Genome Sequencing Producing Precise Diagnoses for Range of Rare Diseases
A Swedish study analyzed 5 years’ worth of data from over 3000 patients, 40% of whom received a molecular diagnosis.
Assessing Novel Treatments to Reduce Thrombosis in PV, ET Requires New Approaches
According to the researchers, few treatments have been able to reduce the thrombotic burden in patients with polycythemia vera (PV) and essential thrombocytopenia (ET), and progress on this front has been hindered by a lack of studies designed to assess a treatment’s impact on thrombotic events.
Gene Expression Profiles May Provide Complementary Prognostic Information for MF
The researchers of the study say their findings may aid the hematological community in determining the value of integrating gene expression profiles in contemporary prognostic models to better identify high-risk patients with a poor prognosis.
The Application of Guideline Adherence Around MPNs
Ruben Mesa, MD, and Jamile M. Shammo, MD, discuss treatment guidelines, pathways, and pharmacologic treatment options that play a role in the standard of care for MPNs.
How Does Race Play a Role in Complications, Survival in MPN?
While the findings of the small study indicate that there was no statistically significant difference in the rate of vascular complications between White and non-White patients with disease, there were differences in survival.
Novel Approach Fusing Top Neck Vertebrae Shows Promise in Bow Hunter Syndrome
Study findings detail the researchers’ ability to prevent stroke in 11 patients ranging in age from 18 months to 15 years
Practice Guidelines and the Decision-Making Process for MPNs
Michael Reff, RPh, MBA, and Kathy Oubre explain the internal guiding documents that aid a practice’s decision-making and workflow when treating patients with MPNs.
Heterogeneity of Therapy in Patients With MPNs
Ruben Mesa, MD, and Jamile M. Shammo, MD, discuss available treatment options, such as JAK inhibitors, and the goals of treatment for patients with MPNs.
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