Rare Blood

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July 18th 2025

CGT Access Model Aims to Expand Access to Sickle Cell Treatments

June 26th 2025

Marstacimab Improved Key Bleeding Outcomes in Hemophilia A or B

May 19th 2025

FDA Approves Jivi for Pediatric Patients Aged 7 to 12 Years With Hemophilia A

March 28th 2025

FDA Approves Fitusiran for Reducing Bleeds in Patients With Hemophilia A/B

December 24th 2024

Research Supports Need for Diversity in Hemophilia Trials and Reducing Resource Strain

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)
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July 18th 2025

How Fitusiran Changes the Treatment Landscape in Hemophilia: Guy Young, MD

June 7th 2024

Ongoing Research in Plasminogen Deficiency Type 1: Dr Amy Shapiro

May 28th 2024

Patients With Plasminogen Deficiency Should Receive Care at Hemophilia Treatment Centers: Dr Amy Shapiro

May 20th 2024

Ryplazim Marks First FDA Approval for Plasminogen Deficiency: Dr Amy Shapiro

May 14th 2024

Ligneous Lesions Often Misdiagnosed in Rare Plasminogen Deficiency: Dr Amy Shapiro

August 19th 2023

Dr Kevin Davies Discusses Challenges of Streamlining CRISPR Technology Delivery

April 20th 2023

Dr David Eagle: Progress in Hematology/Oncology Is Not Without Its Difficulties

More News

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December 23rd 2024

First Subcutaneous Prophylaxis for Hemophilia A and B With Inhibitors Approved by FDA

Giuliana Grossi
The approval of concizumab-mtci (Alhemo) injection marks a significant milestone in managing hemophilia A and B with inhibitors by preventing or reducing bleeding episodes in adults and children 12 years and older.
Top 5 Rare Blood
December 20th 2024

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

Giuliana Grossi
Key findings from 2024 focused on conditions like vaccine-induced immune thrombotic thrombocytopenia, immune thrombocytopenia, and hemophilia, stressing the importance of early detection and innovative therapies.
There is a persistent unmet need for more effective therapies for hemophilia. Image credit: Mayava - stock.adobe.com.jpeg
December 12th 2024

Unmet Needs in Hemophilia: Prophylaxis, Bleeding, Quality of Life

Giuliana Grossi
A pair of studies presented at the 2024 American Society of Hematology (ASH) Annual Meeting & Exposition highlight continuing gaps in hemophilia care despite the availability of prophylactic therapies.
Image of blood for hemophilia analysis-shidlovski-stock.adobe.com.jpeg
December 11th 2024

Rare Blood Disease Innovations Address Unmet Needs, According to ASH Data

Giuliana Grossi
Innovations in rare blood disorders took center stage at the 2024 American Society of Hematology (ASH) Annual Meeting & Exposition, with key research presented on immune thrombocytopenia, hemophilia, and multiple myeloma.
Scientific researcher in a lab-MIND AND I-stock.adobe.com.jpeg
December 6th 2024

New Data Reinforce Iptacopan's Role in Transforming PNH Care With Hemoglobin, QOL Gains

Giuliana Grossi
Promising topline results from a phase 3B study show the efficacy and quality of life (QOL) improvements of iptacopan (Fabhalta) as a twice-daily oral monotherapy for adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who transitioned from anti-C5 therapies.
Young black woman gets advice from senior pharmacist while buying medicine in drugstore - Drazen - stock.adobe.com
December 3rd 2024

Global Burden of Sickle Cell Disease Warrants Universal Access to Hydroxyurea, Experts Say

Giuliana Grossi
Experts are now urging the inclusion of sickle cell disease in the global health agenda to address disparities in treatment access.
Clinical trial results-Egor-stock.adobe.com.jpeg
November 26th 2024

Progression-Free Survival in Relapsed Multiple Myeloma Extended With Ixazomib, Data Show

Giuliana Grossi
Despite challenges such as high attrition rates and adverse events, the trial highlights the value of established therapies in improving outcomes, particularly in regions with limited access to chimeric antigen receptor T cells and bispecific antibodies.
Blood testing - angellodeco - stok.adobe.com.jpg
November 22nd 2024

A Case for Iron Deficiency as a Marker of Inequality in Care Access and Treatment

Giuliana Grossi
Iron deficiency represents more than a medical condition; it is a reflection of persistent health disparities, according to an editorial published in The Lancet Haematology.
Illustration from research for genomic medicine or gene therapy | image credit: RFBSIP - stock.adobe.com.jpeg
November 7th 2024

Next-Gen Hemophilia Treatments Offer Hope for Joint Health, but Accessibility Challenges Remain

Giuliana Grossi
The future of hemophilia care lies not only in technological advancements but also in the pursuit of health equity, ensuring that all patients have access to effective, affordable treatments.
Nurse with protective mask drawing blood for laboratory analysis during coronavirus crisis - Microgen - stock.adobe.com
November 5th 2024

Polycythemia Vera Management: Addressing the Burden of Symptoms and Phlebotomy Dependence

Giuliana Grossi
Although polycythemia vera management remains focused on reducing the risk of thrombotic events, there is growing recognition of the need to address the persistent decline in quality of life experienced by many patients.
Before transfusion dose blood inside hospital room - sudok1 - stock.adobe.com
October 30th 2024

High Health Care Burden of Transfusion-Dependent β-Thalassemia

Giuliana Grossi
Patients with transfusion-dependent β-thalassemia experienced 14.8 hospitalizations annually, according to the findings.
Medicine doctor analysis and diagnosis checking health of patient online and testing result with modern virtual interface on laptop with stethoscope in hand, Online Medical global network - ipopba - stock.adobe.com
October 30th 2024

New AI Tool Identifies Undiagnosed PNH in Health Records

Giuliana Grossi
The machine learning model shows promise in detecting paroxysmal nocturnal hemoglobinuria (PNH) by assessing electronic health records (EHR) data, potentially transforming the diagnostic landscape for rare diseases.
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October 28th 2024

HSSP Pharmacists Demonstrate Substantial Cost Avoidance in Specialty Medication Use

Giuliana Grossi
Cost avoidance varied by clinical setting, with the largest savings occurring in hematology clinics ($73,773), followed by rheumatology ($45,330) and pediatric gastrointestinal/irritable bowel syndrome ($16,519).
hand in rubber gloves holding a hormonal implant - Celso Pupo - stock.adobe.com
October 23rd 2024

Etonogestrel Contraceptive Implants Reduce Pain Crises in Women With Sickle Cell Disease

Giuliana Grossi
Etonogestrel-releasing contraceptive implants in women with sickle cell disease significantly reduced pain intensity and frequency of pain crises over 12 months, with no adverse changes in metabolic or liver function markers.
Hands holding blood, give blood donation, blood transfusion, world blood donor day, world hemophilia day concept - SewcreamStudio - stock.adobe.com
Published: October 16th 2024 | Updated: October 16th 2024

Access Has Improved for Diverse Patients Needing Umbilical Cord Blood Transplant, but Disparities Remain

Giuliana Grossi
Despite advancements in umbilical cord blood transplants, social determinants of health, such as poverty and public insurance, continue to impact outcomes, underscoring the need for equitable access to lifesaving treatments.
Red blood cells | Image credit: Getty Images
October 11th 2024

FDA Approves Marstacimab, First Weekly Sub-Q Option for Hemophilia B

Mary Caffrey
Switching patients with hemophilia B to gene therapy may compound immense health care savings in the long term | image credit: ejgrubbs - stock.adobe.com
September 25th 2024

Gene Therapy for Hemophilia B Could Lower Health Plans’ Budget Over Time

Jared Kaltwasser
Health plans would see savings faster if they switched to etranacogene dezaparvovec sooner, the investigators found.
Sickle cell anemia affects an estimated 100,000 people in the US | image credit: Studios - stock.adobe.com
September 20th 2024

Urinary Angiotensinogen Predicts Persistent Albuminuria in Sickle Cell Anemia

Karen Jacobson-Sive
Patients with sickle cell anemia (SCA) and persistent albuminuria (PA) exhibited significantly higher levels of urinary angiotensinogen (AGT).
Genetic variation and sex-specific differences may be related to pain in SCD | image credit: Lila Patel - stock.adobe.com
September 18th 2024

Pain in SCD: Are Genetic Variations Responsible?

Nameera Temkar, MD
Genetic variability and sex-specific differences may play a role in sickle cell disease (SCD)-related pain; however, more research is needed.
Patients with SCD have reportedly felt stigmatized due to their race, pain, and more | image credit: Andrey Popov - stock.adobe.com
September 11th 2024

Study: Patients With SCD Report Discrimination Based on Race, Pain, and Other Factors

Jared Kaltwasser
A new report based on patient focus groups suggests many clinicians can do more to help patients with sickle cell disease (SCD) feel heard and understood.
Despite treatment, some patients with PNH still experience anemia | image credit: ImageFlow - stock.adobe.com
August 28th 2024

Pegcetacoplan May Reduce Fatigue in PNH Accompanied by Anemia

Kyle Munz
Findings from a post hoc analysis of 3 clinical trials support the switching to or initiation of pegcetacoplan to manage fatigue for patients with paroxysmal nocturnal hemoglobinuria (PNH) that is accompanied by mild to moderate anemia.
Quality of life in patients with hemophilia A or B benefits from the use of concizumab | image credit: Patrick Daxenbichler - stock.adobe.com
August 18th 2024

Patient-Reported Outcomes Favor Concizumab Prophylaxis in Hemophilia A or B

Connor Iapoce
Analysis of the phase 3 explorer7 (NCT04083781) study indicates a preference for concizumab prophylaxis over no prophylaxis in patients with hemophilia A or B with inhibitors.
Patients with PNH may become more active following ravulizumab therapy | image credit: sutadimages - stock.adobe.com
August 16th 2024

Ravulizumab Boosts Activity Levels, Quality of Life in Patients With PNH

Connor Iapoce
Ravulizumab therapy may enable patients with paroxysmal nocturnal hemoglobinuria (PNH) to comparable achieve activity levels and patient-reported outcomes to the US general population.
Sickle cell retinopathy is a manifestation of SCD that occurs in the eye and can cause detriments to patients’ vision| image credit: Катерина Євтехова - stock.adobe.com
August 5th 2024

The Benefits of More Sickle Cell Retinopathy Screening for Patients With SCD

Kyle Munz
A systematic analysis that reviewed screening for sickle cell retinopathy in patients with sickle cell disease (SCD) underscores the importance of advanced imaging measures and further research on screening implementation methods.
COVID-19 vaccine injection | image credit: insta_photos - stock.adobe.com
July 31st 2024

COVID-19 Vaccination Shows Favorable Risk-Benefit in Sickle Cell

Connor Iapoce
A study from the American Society of Hematology (ASH) Research Network reports positive antibody response and tolerability after mRNA vaccination in people with sickle cell disease.
Efanesoctocog alfa is a high-sustained factor VIII replacement therapy for the treatment of hemophilia A | image credit: Ekaterina - stock.adobe.com
July 25th 2024

Efanesoctocog Alfa Prophylaxis Promising for Pediatric Severe Hemophilia A

Kyle Munz
The phase 3, international, open-label, single-group XTEND-Kids study concluded that children with severe hemophilia A younger than 12 years benefited from treatment with efanesoctocog alfa prophylaxis.
Hemolysis is the destruction of red blood cells, a common symptom of PNH | image credit: Red Lemon - stock.adobe.com
July 22nd 2024

Risk Mitigation for Hemolysis Is Needed During PNH Treatment, Study Finds

Kyle Munz
The phase 3 PEGASUS trial found that patients with paroxysmal nocturnal hemoglobinuria (PNH) may still experience anemia following recommended therapies, underscoring the need for further research.
Fatigue is one of the most prevalent non-painful symptoms reported in SCD | image credit: Feng Yu - stock.adobe.com
July 20th 2024

Pain Threshold Predictive of Fatigue Severity in SCD, Study Finds

Kyle Munz
A cross-sectional study found that static pain measures could be useful for predicting fatigue severity in patients with sickle cell disease (SCD).
Clotting complications are a severe symptom of TTP | image credit: Artur - stock.adobe.com
July 15th 2024

Patients With ADAMTS13 Deficiency and/or TTP Experience High Clinical Burden

Kyle Munz
A retrospective database analysis demonstrated the high clinical burden experienced by patients with severe deficiencies of ADAMTS13 and thrombotic thrombocytopenic purpura (TTP)–related diagnoses.
Children and adults with SCD can exhibit various cognitive deficits or neurological injury that could be related to their disease | image credit: Werckmeister - stock.adobe.com
July 9th 2024

Cognitive Health Unaffected by Diluted Perivascular Spaces in Children With SCD

Kyle Munz
An investigation into the burden of dilated perivascular spaces (dPVS) in children with sickle cell disease (SCD) confirmed that those with SCD experience more severe manifestations of dPVS compared with the general population.
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