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The first gene therapies for sickle cell disease were FDA approved in 2023, but high upfront costs have been barriers to patient access to these life-changing treatments. | Image credit: RFBSIP - stock.adobe.com
CGT Access Model Aims to Expand Access to Sickle Cell Treatments

July 18th 2025

States participating in the Cell and Gene Therapy (CGT) Access Model will be testing outcomes-based payments for sickle cell disease treatments.

Hemophilia | Image credit: jarun011 - stock.adobe.com
Marstacimab Improved Key Bleeding Outcomes in Hemophilia A or B

June 26th 2025

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

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

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

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.”
Platelets forming a blood clot, AI Generative: © Катерина Євтехова - stock.adobe.com
April 16th 2023

Religious Limitations Likely Not to Preclude Successful TTP Therapy

Jared Kaltwasser
A review of cases involving Jehovah’s Witnesses found many modalities can be successfully deployed to treat thrombotic thrombocytopenic purpura (TTP).
Kryotherapie: © CrazyCloud - stock.adobe.com
April 11th 2023

ADAMTS-13 Clearance Major Pathogenic Mechanism in iTTP

Jared Kaltwasser
The findings suggest recombinant ADAMTS-13 may be a useful adjunct therapy to plasma exchange among patients who have thrombotic thrombocytopenic purpura (iTTP).
Doctor writing a medical prescription in hospital: © Kamon Wongnon - stock.adobe.com
April 8th 2023

Case Report Highlights Hurdles in Identifying Pseudo-TTP

Jared Kaltwasser
The patient’s thrombotic thrombocytopenic purpura (TTP)–like symptoms were caused by deficiencies of vitamins B1 and B12.
Thrombotic thrombocytopenic purpura (ttp) on chalk board: © AKDIM_DESIGN - stock.adobe.com
April 4th 2023

iTTP Marked by Reversible Skin Microvascular Hyporeactivity, Study Finds

Jared Kaltwasser
The impaired reactivity may play a role in organ injury, the authors suggested, among patients who have immune-mediated thrombotic thrombocytopenic purpura (iTTP).
Caplacizumab single-variable-domain antibody drug: © molekuul.be - stock.adobe.com
March 31st 2023

“Rational” Approach to Caplacizumab Therapy in TTP Outlined in New Article

Jared Kaltwasser
Judicious use of caplacizumab (Cablivi) can reduce the risk of severe bleeding and help manage costs associated with the therapy in patients living with thrombotic thrombocytopenic purpura (TTP).
Anxiety word cloud with abstract background: © ibreakstock - stock.adobe.com
March 28th 2023

Symptoms of Anxiety, PTSD Present Years After Acute iTTP, aHUS Episodes

Jared Kaltwasser
New data underscore the long-term implications of immune-mediated thrombotic thrombocytopenic purpura (iTTP) and atypical hemolytic-uremic syndrome (aHUS), both rare diseases.
Image representing different life expectancies: Hyejin Kang - stock.adobe.com
March 24th 2023

Sickle Cell Disease Cuts 20 Years From Life Expectancy, Study Finds

Jared Kaltwasser
People with the inherited disease can expect to live 52.6 years following birth, the report shows.
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March 20th 2023

TCD Screening and Spending Among Children With Sickle Cell Anemia

Sarah L. Reeves, PhD Sophia Ng, PhD Kevin J. Dombkowski, DrPH Jean L. Raphael, MD Kao-Ping Chua, MD, PhD
A substantial proportion of families of privately insured children with sickle cell anemia pay more than $100 for essential stroke screenings, a high-value service.
Lab tech holding blood samples: Africa Studio - stock.adobe.com
March 18th 2023

Validation Study Affirms Prognostic Value of MITS in TTP

Jared Kaltwasser
Investigators said the Mortality in TTP Score (MITS) was effective at characterizing the risk of death in patients hospitalized with thrombotic thrombocytopenic purpura (TTP).
Image of a lab tech with a test tube labelled "anemia": vchalup - stock.adobe.com
March 14th 2023

Treatment of Rare Anemias Shifting Rapidly

Jared Kaltwasser
Investigators said several new anemia therapies are in development, and many of them may help wide swaths of patients.
FDA Approves Efanesoctocog Alfa for Hemophilia A
February 24th 2023

FDA Approves Efanesoctocog Alfa for Hemophilia A

Allison Inserro
Efanesoctocog alfa is the first factor VIII therapy to overcome the interaction with endogenous von Willebrand factor, which creates a ceiling of 8 to 19 hours on the half-life of current factor VIII replacement products, which in turn creates the need for more frequent dosing.
image of newspapers and coffee on a table with text "What We're Reading: Your top daily health care news from AJMC®"
January 17th 2023

What We’re Reading: Sickle Cell Cure Complexity; Free Care for Veterans in Suicidal Crisis; Pfizer to Offer Low-Cost Drugs to Underserved Countries

AJMC Staff
The reality of a sickle cell disease cure brings fear, optimism, and questions to patients’ lives; veterans who are in suicidal crisis can receive free emergency care at any Department of Veterans Affairs or private facility; Pfizer to increase access to progressive treatments through sale of drugs at non-profit prices to poor countries.
Top 5 Most-Read Rare Blood Content of 2022
December 22nd 2022

Top 5 Most-Read Rare Blood Content of 2022

Pearl Steinzor
The most-read rare blood articles included studies on cold agglutinin disease , hemophilia B gene therapy, autoimmune hemolytic anemia, and hereditary thrombotic thrombocytopenia purpura.
Image of blood in a test tube
December 22nd 2022

Conquering SCD by Confronting Treatment Barriers, Investigating PROs

Maggie L. Shaw
A pair of abstracts presented during this year’s 64th American Society of Hematology Annual Meeting and Exposition explored barriers to treatment for sickle cell disease (SCD).
Image of blood flowing
December 20th 2022

Analysis of Beti-Cel for Severe β-Thalassemia Shows Extensive, Long-term Patient Improvement

Maggie L. Shaw
A pair of abstracts presented at this year’s 64th American Society of Hematology Annual Meeting and Exposition bear out the significant transfusion-free rate at 3 years following beti-cell administration and marked improvements in patient-reported outcomes, including the ability to work and be physically active.
Blood
December 9th 2022

Phase 3 CADENZA Trial Validates QOL Improvements Reported by Patients With CAD Receiving Sutimlimab

Pearl Steinzor
Mental and physical health aspects of quality of life (QOL) improved after sutimlimab treatment in patients with cold agglutin disease (CAD) during a phase 3 trial.
Image of a doctor and patient talking
November 18th 2022

Some Patients With CAD Experience Sustained Remission After Sutimlimab Discontinuation

Rose McNulty
A research letter published in Blood Advances details the first clinical evidence of sustained hematologic remission for cold agglutinin disease (CAD) after sutimlimab treatment discontinuation.
Child in exam room
November 14th 2022

Study Highlights Value of PROs in Children With Sickle Cell Disease During Acute Pain Episodes

Rose McNulty
Sufficiently powered studies are necessary to investigate associations between clinical measures and patient-reported outcomes (PROs) in children with sickle cell disease experiencing acute pain episodes, according to a recent study.
Teddy Bear
November 12th 2022

Report Suggests Best Practices for Care of Pediatric Sickle Cell Disease

Rose McNulty
A panel of specialists proposed 26 essential elements for comprehensive pediatric sickle cell disease care, laying groundwork for standardized guidelines and the establishment of accredited care centers in the future.
Image of sickled cells
November 11th 2022

Real-World Study of Voxelotor for Sickle Cell Disease Shows Safety, Efficacy Similar to Clinical Trials

Rose McNulty
Over the course of voxelotor treatment, hemoglobin levels and hemolysis markers improved, with results sustained over the treatment period.
image of high health care costs
November 7th 2022

Sickle Cell Disease With Recurrent Vaso-Occlusive Crises Carries a Substantial Economic Burden, Study Finds

Rose McNulty
The analysis emphasizes the association between vaso-occlusive crisis frequency and health care costs, which are inflated largely due to inpatient visits.
Picture of red cells
October 27th 2022

Case Report Describes Thrombocytopenia Secondary to Iron-Deficiency Anemia

Rose McNulty
A recently published report describes the diagnosis and treatment of a case of iron-deficiency anemia–associated thrombocytopenia secondary to recurrent nosebleeds.
graphic depicting patient data
October 21st 2022

Improved Surveillance, Registry Data Could Improve Outcomes in Sickle Cell Disease

Rose McNulty
Population-based data is a crucial aspect to evaluating current care quality and improving outcomes for patients with sickle cell disease.
Blood cells
October 15th 2022

Review Addresses Proper Applications of Sutimlimab in Cold Agglutinin Disease

Rose McNulty
A review published in the journal Immunotherapy summarizes the applications of sutimlimab and emphasizes the importance of individualizing treatment for patients with cold agglutinin disease.
Sickle cells
October 12th 2022

Sickle Cell Disease Management May Overlook Impactful Complications, Study Finds

Rose McNulty
Findings of a recent study suggest that complications of sickle cell disease (SCD) significantly contribute to morbidity and are more prevalent among older patients.
Image of a happy life
October 3rd 2022

Sutimlimab Improves Patient-Reported QOL in Cold Agglutinin Disease

Rose McNulty
An analysis of patient-reported outcomes from the pivotal CARDINAL trial of sutimlimab in cold agglutinin disease found that quality of life (QOL) improved and persisted throughout treatment.
Blood cells
October 1st 2022

Review: More Research on Treatment for Extramedullary Hematopoiesis in Transfusion-Dependent Beta Thalassemia Is Needed

Rose McNulty
Currently, there are no clear guidelines for managing extramedullary hematopoiesis in transfusion-dependent beta thalassemia.
Image of a sick child
September 26th 2022

Guidance for Evaluating for Bleeding Disorders in Suspected Child Abuse

Rose McNulty
The recommendations aim to make this type of evaluation more consistent in cases when children have suspicious bruising or bleeding patterns.
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