Rare Blood

Latest News

hemophilia and kids | Image credit: Denira - stock.adobe.com
FDA Approves Jivi for Pediatric Patients Aged 7 to 12 Years With Hemophilia A

May 19th 2025

The FDA previously approved Jivi in 2018 to treat adults aged 12 years and older with hemophilia A.

FDA approval-Olivier Le Moal-stock.adobe.com
FDA Approves Fitusiran for Reducing Bleeds in Patients With Hemophilia A/B

March 28th 2025

hemophilia-jarun011-stock.adobe.com
Research Supports Need for Diversity in Hemophilia Trials and Reducing Resource Strain

December 24th 2024

FDA-Deni-stock.adobe.com
First Subcutaneous Prophylaxis for Hemophilia A and B With Inhibitors Approved by FDA

December 23rd 2024

Top 5 Rare Blood
Top 5 Most-Read Articles on Rare Blood Disorders in 2024

December 20th 2024

Treating PNH: Complement Inhibitors, Administration Modes, and Coordinated Care Needs

Join experts as they explore PNH pathophysiology, treatment strategies, clinical trials, and management challenges. Gain essential insights to enhance patient outcomes and optimize PNH treatment.

View the latest AJMC Peer Exchange on paroxysmal nocturnal hemoglobinuria (PNH)

More News

Brain
August 15th 2023

Pain, Fatigue Not Associated With Neurocognitive Performance in Adolescents With SCD

Julia Bonavitacola
In a study of adolescents and young adults with sickle cell disease (SCD), researchers did not find a link between neurocognitive performance and pain and fatigue.
Pain meter
August 9th 2023

SCD Pain Impact Associated With Social, Emotional Functioning

Julia Bonavitacola
The pain and impact of sickle cell disease (SCD) was associated with patient social and emotional functioning in a recent study.
Blood cells
August 1st 2023

Caplacizumab Effective in Treating aTTP Along With Standard Regimen

Julia Bonavitacola
Patients with acquired thrombotic thrombocytopenic purpura (aTTP) showed significant benefits in critical outcomes when treated with caplacizumab as well as the standard regimen.
Man writing words Vitamin B12 on chalkboard: © Stepan Popov - stock.adobe.com
July 30th 2023

Vitamin B Deficiency Can Mimic TTP, Case Report Warns

Jared Kaltwasser
By giving their patient the correct diagnosis, clinicians avoided costlier treatments for thrombotic thrombocytopenic purpura (TTP) like plasmapheresis.
One person is answering question about thrombotic thrombocytopenic purpura: © Richelle
July 27th 2023

Study Validates First Disease-Specific PRO Instrument for Patients With Congenital TTP

Jared Kaltwasser
Congenital thrombotic thrombocytopenic purpura (TTP), an ultra-rare disease, can significantly impact quality of life, but clinicians have previously lacked a disease-specific way to track experiences via patient-reported outcomes (PROs).
Blood and DNA in test tube
July 24th 2023

Case Report Highlights Diagnostic, Treatment Challenges of Concurrent TTP, RCC

Jaime Rosenberg
The researchers noted that their paper is the first, to their knowledge, to report on a case of thrombocytopenic purpura (TTP) in a patient with metastatic renal cell carcinoma (RCC).
Health care concept about Osteomyelitis with inscription on the sheet: © Yurii Kibalnik - stock.adobe.com
July 20th 2023

Osteomyelitis Masked as Apparent Case of Vaso-Occlusive Pain in Child With SCD

Jared Kaltwasser
Study authors say the consequences of misdiagnosing osteomyelitis can be severe in the disease setting of sickle cell disease (SCD).
uterine bleeding word or concept represented by black and white letter cubes on a grey horizon background stretching to infinity: © lexiconimages - stock.adobe.com
July 14th 2023

Abnormal Uterine Bleeding May Be Caused by Clotting Factor Deficiencies

Jared Kaltwasser
A new review article illuminates why physicians should keep coagulation disorders in mind when evaluating patients with heavy abnormal uterine bleeding.
Young african american Boy in Hospital: © WavebreakMediaMicro - stock.adobe.com
July 11th 2023

Neurodevelopmental Complications of SCD Exacerbated by SDOH

Jared Kaltwasser
Black children with sickle cell disease (SCD) face a disproportionate burden of social determinants of health (SDOH), the study found.
pain: © boygostockphoto - stock.adobe.com
July 8th 2023

Research Explores Factors Linked With SCD Pain Severity, Frequency

Gianna Melillo
Investigators stress the importance of ongoing research on the association between depression and pain in patients with sickle cell disease (SCD).
Array of blood samples for microscopy and biopsy tissue on blue: © motorolka - stock.adobe.com
July 5th 2023

Review Assesses Role of Liquid Biopsies in Myeloid Malignancies

Gianna Melillo
Artificial intelligence may be leveraged to improve minimal residual disease measurements for patients.
BioMarin
June 29th 2023

BioMarin Receives Approval for First Gene Therapy for Adults With Severe Hemophilia A

Mary Caffrey
Approximately 2500 individuals in the United States could be eligible to receive valoctocogene roxaparvovec.
Laboratory doctor or scientific researcher preparing for a smear test or blood film. Using a pipette the doctor will put a drop of blood on the sterile slide See Less: © raresb - stock.adobe.com
June 28th 2023

Unique Giant Cancer Cells Found in Patients With Myelodysplastic Syndromes

Gianna Melillo
Authors suggest the finding may indicate the cells play a role in hematological malignancies.
Pilot Program Aims to Boost Population-Level Data on Sickle Cell Disease
June 24th 2023

Pilot Program Aims to Boost Population-Level Data on Sickle Cell Disease

Gianna Melillo
The infrastructure also provides a blueprint for similar initiatives in other rare diseases.
Kidneys
June 23rd 2023

Review Assesses Current, Emerging Therapies for Kidney Manifestations in SCD

Gianna Melillo
A recent review highlighted a selection of emerging therapies and advances in sickle cell disease (SCD) that focus on renal manifestations.
Medical concept meaning Myelodysplastic syndrome with inscription on the piece of paper: © Yurii Kibalnik - stock.adobe.com
June 14th 2023

Research Suggests Diagnostic Evaluation May Be of Little Value to Older Patients With MDS

Gianna Melillo
The study also highlighted disparities in accurate diagnosis of myelodysplastic syndromes (MDS).
Woman scratching her arm: © thodonal - stock.adobe.com
June 10th 2023

Study Underscores Need for Multidisciplinary Management of Skin Rashes in Patients Completing HCT

Gianna Melillo
Skin rashes are common among patients who complete hematopoietic cell transplantation (HCT).
basic science image
June 8th 2023

Roxadustat Misses Mark as Anemia Treatment in Transfusion-Dependent, Lower-risk MDS

Kristi Rosa
The phase 3 MATTERHORN trial examining roxadustat as an anemia treatment in patients with transfusion-dependent, lower-risk myelodysplastic syndrome did not meet its primary end point.
neoplasm word on grey background: © chrupka - stock.adobe.com
June 3rd 2023

Researchers Assess Effects of Updated Myeloid Neoplasm Classifications

Gianna Melillo
The new classifications included several major changes and may help identify different subtypes.
Scientist pipetting sample into a vial for DNA testing: © Cavan - stock.adobe.com
June 2nd 2023

Case Study Highlights Importance of Next-Generation Sequencing in Patients With ET

Gianna Melillo
To the authors’ knowledge, the study is the first to include a patient with essential thrombocythemia (ET) and MPL and Type 1 calreticulin gene mutations.
Medical concept about Sickle Cell Disease with sign on the sheet: © Yurii Kibalnik - stock.adobe.com
May 20th 2023

Stroke Management in SCD Requires Vigilance, Careful Monitoring

Jared Kaltwasser
Investigators said disease-modifying therapies may change the treatment landscape of sickle cell disease (SCD).
multitasking in hands: © Romolo Tavani - stock.adobe.com
May 17th 2023

Patients With SCD Prefer Digital CBT if It’s Personalizable and Social

Jared Kaltwasser
Focus groups show patients who have sickle cell disease (SCD) like the idea of a digital health app for cognitive behavioral therapy (CBT) that allows them to make meaningful connections with people with shared experiences.
Hematoma word cloud: © ibreakstock - stock.adobe.com
May 13th 2023

Case Report Shows Hematomas Can Appear in Unexpected Places

Jared Kaltwasser
The patient had hemophilia A, but reported no trauma preceding the hematoma.
Young person holding hologram projection displaying health related graphs and symbols: © ra2 studio - stock.adobe.com
May 9th 2023

People With Sickle Cell Disease Willing to Share Data as Partners, Not “Specimens”

Jared Kaltwasser
The results of a new focus group offer insights on how and when people with rare diseases are willing to share their personal health information.
vials of blood
May 4th 2023

Iptacopan Meets Primary End Point of APPOINT-PNH Trial in Paroxysmal Nocturnal Hemoglobinuria

Caroline Seymour
Iptacopan elicited improved hemoglobin levels measuring at least 2 g/dL higher vs baseline, leading to transfusion independence after 24 weeks in approximately 92.2% of patients with complement inhibitor–naïve paroxysmal nocturnal hemoglobinuria.
Colorful DNA molecule. Concept image of a structure of the genetic code: © ktsdesign - sotck.adobe.com
May 2nd 2023

Gene Editing, mRNA Hold Promise as Fanconi Anemia Therapies

Jared Kaltwasser
Gene therapies could have a major impact, although the cost and complexities could inhibit their wide rollout, study authors said.
Acute coronary syndrome (heart disorder) diagnosis medical concept on tablet screen with stethoscope: © ibreakstock - stock.adobe.com
April 27th 2023

Acute Coronary Syndrome Increases Risk of In-Hospital Mortality in TTP

Jared Kaltwasser
Investigators found patients with acute coronary syndrome were 3 times more likely to die in the hospital.
Brain Volume May Serve as Biomarker of Brain Injury in Patients With Sickle Cell Disease
April 26th 2023

Brain Volume May Serve as Biomarker of Brain Injury in Patients With Sickle Cell Disease

Gianna Melillo
The data highlight that silent cerebral infarcts represent just a portion of brain injury that occurs in patients with sickle cell disease (SCD), and brain volume can serve as another potential biomarker of brain injury in those with the disease.
Man taking medicaation: © Rawpixel.com - stock.adobe.com
April 23rd 2023

High Use of Antidepressants, Anxiety, Pain Meds Seen Among Patients With Hemophilia

Gianna Melillo
These findings underscore the need for improved bleed protection and hemophilia care for those with all severities of the disease.
Joseph Alvarnas, MD, Discusses New CMS Drug Pricing Models, Impact for Blood Diseases
April 19th 2023

Joseph Alvarnas, MD, Discusses New CMS Drug Pricing Models, Impact for Blood Diseases

Interview by Mary Caffrey
CMS in February 2023 announced 3 new models for testing by the Center for Medicare & Medicaid Innovation—all of which “aim to lower the cost of drugs, promote accessibility to life-changing drug therapies, and improve quality of care.”
<1234567>

2 Commerce Drive
Suite 100
Cranbury, NJ 08512

© 2025 MJH Life Sciences® and AJMC®.
All rights reserved.

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