Rare Disease

Latest News

Neoplasms, cancerous disease, malignant tumour | Ирина Батюк - stock.adobe.com
NCCN Guidelines Update Adds Momelotinib Below Ruxolitinib for High-, Low-Risk Myelofibrosis

January 23rd 2024

Momelotinib was given category 2A and 2B status for patients with high- and low-risk myelofibrosis (MF) and MF with anemia. However, ruxolitinib retains a higher category of recommendation as a treatment for patients with MF.

Top 5 Most-Read Rare Disease Articles of 2023
Top 5 Most-Read Rare Disease Articles of 2023

December 29th 2023

sickle cell disease | Image credit: Dr_Microbe - stock.adobe.com
Interventions Needed to Increase DMT Uptake in Sickle Cell Disease

December 26th 2023

Polycythemia vera, a rare slow-growing blood cancer with an increase in the number of red blood cells | Dr_Microbe - stock.adobe.com
Age-Adjusted Index, Machine Learning Are Significant Factors for Predicting Thrombosis in Polycythemia Vera

December 18th 2023

Matthew Frigault, MD, MS, Harvard Medical School, Massachusetts General Hospital Cancer Center
Novel BCMA-Directed CAR Anito-Cel Is Highly Effective for Myeloma

December 12th 2023

Exploring the Implementation of Quality Care Programs for Patients with Myeloproliferative Neoplasms

Bruce Feinberg, DO; Kathy Oubre, Michael Reff, RPh, MBA; Jamile M. Shammo, MD; and Ruben Mesa, MD, discuss the incorporation of quality care programs for improved disease management and patient care for MPNs.

Exploring the Implementation of Quality Care Programs for Patients with Myeloproliferative Neoplasms peer exchange

More News

coffee and newspaper
March 1st 2023

What We’re Reading: Rare Disease Drug Approved; Congress and PBMs; FDA Panel Splits on RSV Shot

AJMC Staff
The FDA approved the first drug for the rare disease Friedreich’s ataxia; House Republicans seek information from pharmacy benefit managers (PBMs); FDA panel narrowly supports respiratory syncytial virus (RSV) vaccine for older adults.
On Rare Disease Day, PKD Foundation, IQVIA Highlight Patient Registry Gathering Quality-of-Life Data
February 28th 2023

On Rare Disease Day, PKD Foundation, IQVIA Highlight Patient Registry Gathering Quality-of-Life Data

Mary Caffrey
The Polycystic Kidney Disease Foundation and IQVIA are partners in the development of the ADPKD Registry, which is tracking longitudinal quality-of-life information from patients across North America.
Image of blood being tested in a lab
February 27th 2023

Masked PV Case Highlights Challenge of Diagnosis in Younger Women

Jared Kaltwasser
Features of polycythemia vera (PV) can easily be misattributed to other causes, such as iron depletion or arterial hypertension.
Image of patients of different races
February 23rd 2023

Similar Survival Rates Seen Among Black African Americans With PMF, Other Groups

Jared Kaltwasser
The study also found, however, that transplant outcomes appear to be worse for Black African American patients who have primary myelofibrosis (PMF), although the study utilized a small sample size.
graphic depicting survival
February 20th 2023

Spleen Response Closely Linked With Survival in MF While on Ruxolitinib

Jared Kaltwasser
Patients with myelofibrosis (MF) who achieved a spleen response while taking ruxolitinib and lost it had survival outcomes similar to patients who did not respond, according to results from a real-world study.
Image of scientific research
February 18th 2023

Ruxolitinib Well Tolerated in Untreated Patients With Polycythemia Vera

Jared Kaltwasser
The results of the futility analysis also suggest the therapy leads to significant clinical benefits.
Image of a doctor reading a case report
February 14th 2023

Two Cases Demonstrate Potential Renal Complications of MDS/MPN

Jared Kaltwasser
Evidence suggests as many as 29% of people with myeloproliferative neoplasms (MPN) or myelodysplastic syndrome (MDS)/MPN have chronic kidney disease.
Genetic Screening Could Have Promise for Reducing Infant Deaths
February 11th 2023

Genetic Screening Could Have Promise for Reducing Infant Deaths

Julia Bonavitacola
An analysis of 112 infant deaths found that the contribution of genetic diseases to mortality was higher than previously known, and that treatments are available for 30% of the genetic diseases uncovered by the study.
graphic depicting children
February 3rd 2023

Unmet Psychosocial Needs of Caregivers Impacted HRQOL for Children With Rare Diseases During the Pandemic

Justina Petrullo
At the start of the COVID-19 pandemic, high levels of distress, overwhelm with daily problems, and unmet psychosocial information needs decreased caregiver-reported health-related quality of life in children with rare diseases.
Doctor using clipboard.
January 28th 2023

Case Report on Portal Vein Thrombosis Linked With Pregnancy, Iron Deficiency Anemia After C-Section in Patient With PV

Matthew Gavidia
Researchers explored the case of a 40-year-old woman with polycythemia vera (PV) who developed a portal vein occlusion 1.5 months postpartum after C-section.
Hospital patient.
January 26th 2023

Bleeding Risk More Than Doubled for AMI Hospitalization With ET

Matthew Gavidia
High bleeding risk was seen among patients hospitalized with acute myocardial infarction (AMI) who had essential thrombocythemia (ET), particularly those treated with percutaneous coronary intervention.
Roger M. Lyons, MD, FACP
January 25th 2023

Roger M. Lyons, MD, Discusses 10-Year Review of MDS Patient Data From the Community Oncology Setting

Interview by Mary Caffrey
Evidence-Based Oncology™️ spoke with Lyons about the creation of this real-world data set and the pros and cons of different treatments for MDS.
Image of genetic sequencing
January 23rd 2023

Low-risk MDS Management May Change as New Technology, Therapies Emerge

Jared Kaltwasser
A new review article shows how technologies like next-generation sequencing might help clinicians tailor care for myelodysplastic syndromes (MDS).
Image of laboratory testing
January 10th 2023

Secondary ADAPT Analysis Shows HRQOL Benefit of Efgartigimod in Myasthenia Gravis

Jaime Rosenberg
Data from the secondary analysis show that the improvements seen in health-related quality of life (HRQOL) translated into clinical improvements.
Top 5 Rare Disease Articles of 2022
December 23rd 2022

Top 5 Most-Read Rare Disease Articles of 2022

Justina Petrullo
The most-read rare disease articles of the year included the topics of new pediatric central nervous system tumor guidelines, graft versus host disease, the contribution of gene variants to pediatric neuropsychiatric conditions, and hope for patients with blast-phase chronic myeloid leukemia.
Image of human body
December 19th 2022

Stiff Person Syndrome Spectrum Disorders: Knowledge Gaps and Future Research Considerations

Rose McNulty
Although rare, stiff person syndrome spectrum disorders can significantly affect patient quality of life, and current knowledge gaps warrant further research.
graphic depicting clinical trial
December 13th 2022

Real-world Study Confirms Clinical Benefit of Ruxolitinib in Heavily Pretreated cGVHD

Rose McNulty
A multicenter study supports the use of ruxolitinib in steroid-refractory and steroid-dependent chronic graft-versus-host disease, adding to promising clinical trial findings.
picture of an ICU
December 12th 2022

Report Details Severe Case of ARDS Following Ruxolitinib Discontinuation

Rose McNulty
The report is the first recorded case of acute respiratory distress syndrome (ARDS) that required venovenous extracorporeal membrane oxygenation and was complicated by spontaneous spleen rupture.
Image of clinical research
November 9th 2022

Need for More Research Into Chronic GVHD After Allogeneic HSCT Highlighted in New Review

Rose McNulty
Although the understanding of chronic graft-versus-host disease (GVHD) has improved in recent years, more work is needed to facilitate personalized treatments for patients after hematopoietic stem cell transplantation (HSCT).
Image of the blood
November 1st 2022

Non–Driver Gene Mutations May Hold Predictive Value in ET and PV

Rose McNulty
A recent study supports the notion that non–driver mutations in essential thrombocythemia (ET) and polycythemia vera (PV) have predictive value and found that a proposed international prognostication model may be useful for Japanese patients.
stethoscope on question mark
October 21st 2022

Clarifying Different Characteristics of Erythrocytosis

Rose McNulty
JAK2-unmutated erythrocytosis has a variety of etiologies, and identifying the underlying cause may prove helpful in cases where it is secondary to another condition such as polycythemia vera.
Stem Cells Highlighted as Potential Therapeutic Option in Huntington Disease
October 15th 2022

Stem Cells Highlighted as Potential Therapeutic Option in Huntington Disease

Rose McNulty
Although no pharmacological treatment offers long-term efficacy or extends survival with Huntington disease at this point, research suggests that stem-cell therapy holds promise in this and other neurodegenerative diseases.
Fatigued man
October 12th 2022

Patients With Lower-Risk MF, Low- or High-Risk ET Report Significant QOL Impacts

Rose McNulty
Despite the use of therapies to address the clinical manifestations of myelofibrosis (MF) and essential thrombocythemia (ET), the symptom burdens of these conditions continue to negatively affect patient quality of life (QOL).
thrombosis
October 8th 2022

Diagnostic and Treatment Considerations for Splanchnic Vein Thrombosis in MPN

Rose McNulty
The review highlighted key diagnostic and treatment considerations for patients with splanchnic vein thrombosis and myeloproliferative neoplasms.
Site Logo
October 5th 2022

Scenario Analysis When Conducting Budget Impact Analyses for Rare Diseases

Eric P. Borrelli, PhD, PharmD, MBA R. Scott Leslie, PhD, MPH
The authors propose conducting a scenario analysis for interventions to treat rare diseases by varying health plan size to demonstrate the variability of potential budget impact.
image of a patient
September 30th 2022

How the Name Change to Myeloproliferative Neoplasms Affected People With the Disease

Leah Lawrence
A decision to rename myeloproliferative neoplasms led to a plethora of developments in a space where there was once little interest.
graphic of brain
September 19th 2022

bluebird bio Receives FDA Accelerated Approval for Rare Brain Disease Gene Therapy

AJMC Staff
The gene therapy is the first treatment for active cerebral adrenoleukodystrophy, a fatal neurodegenerative disease that affects boys.
Bone marrow blast cells
September 13th 2022

Early Timing, Optimal Conditioning Maximize Outcomes of Allo-SCT in MDSs and MPNs

Rose McNulty
A review published in Frontiers in Oncology explored the application of allo-SCT for myelodysplastic syndromes and myeloproliferative neoplasms.
What We're Reading
September 8th 2022

What We’re Reading: TX Judge Rules Against ACA PrEP Coverage; FDA Advisers Recommend ALS Drug Approval; Walmart, UnitedHealth Partnership

AJMC Staff
A Texas judge ruled that HIV pre-exposure prophylaxis (PrEP) coverage under the Affordable Care Act is unconstitutional; an FDA advisory panel recommended the approval of an experimental drug for amyotrophic lateral sclerosis (ALS); Walmart and UnitedHealth Group are collaborating to provide preventive health care for older Americans.
Group of young adults
September 5th 2022

Guidelines Needed for Managing MPNs in AYA Patients, Review Says

Rose McNulty
The management of myeloproliferative neoplasms (MPNs) in adolescent and young adult (AYA) patients is currently similar to that of older patients. However, comprehensive research is needed to form guidelines for this younger population.
<12345678
...
30>

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

© 2024 MJH Life Sciences® and AJMC®.
All rights reserved.

About
Contact
Editorial Boards
Advertise
Do Not Sell My Information
Terms & Conditions
Privacy Policy
CH LogoCenter for Biosimilars Logo