Rare Blood

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December 23rd 2024

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

By Giuliana Grossi

December 20th 2024

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

By Giuliana Grossi

December 12th 2024

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

By Giuliana Grossi

December 11th 2024

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

By Giuliana Grossi

December 6th 2024

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

By Giuliana Grossi

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)

Video Interviews

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

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

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

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

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

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

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

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

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

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

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

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

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.

Patients with PNH stand at an increased risk for thrombotic, among other, events | image credit: Bipul Kumar - stock.adobe.com

June 30th 2024

Crovalimab Offers Effective, Less Burdensome Alternative to Eculizumab for PNH Treatment

For patients with paroxysmal nocturnal hemoglobinuria (PNH), crovalimab has emerged as an additional treatment option with a similar safety and efficacy profile to eculizumab,

The fertility risks that accompany SCD are not completely understood by impacted patients | image credit: Prostock-studio - stock.adobe.com

June 28th 2024

Fertility Risks and Treatments Misunderstood by Patients With SCD

Results from a cross-sectional study revealed the gaps in knowledge related to fertility risks among patients with sickle cell disease (SCD), as well as shed light on patient perspectives on fertility treatments.

Data shows that pregnant individuals have 3 times the risk of contracting COVID vs the general population | image credit: Evgeniya Primavera - stock.adobe.com

June 20th 2024

Sickle Cell Trait Can Exacerbate COVID-19 Risk During Pregnancy

Sickle cell trait (SCT) and pregnancy each exacerbate the symptoms of COVID-19. As a result, pregnant individuals with SCT may have a higher risk of COVID-19 infection.

World Sickle Cell Awareness Day was established on June 19, 2009 | image credit: SaturnO_27 - stock.adobe.com

June 19th 2024

World Sickle Cell Awareness Day: Navigating the Present and Shaping the Future of Sickle Cell Disease

Kyle Munz Giuliana Grossi

June 19 is marked as World Sickle Cell Awareness Day, granting the opportunity to reflect on the current state of treatment, access, equity, and more that impacts patients living with sickle cell disease.

Blood clot forming | image credit: Tatiana Shepeleva - stock.adobe.com

June 5th 2024

Prophylaxis With Recombinant ADAMTS13 Demonstrates Superiority vs Standard Therapy in Congenital TTP

The multinational, open-label, controlled, randomized, crossover, phase 3 TAK-755 (NCT03393975) trial demonstrated superior efficacy and safety of recombinant ADAMTS13 as prophylaxis in patients with congenital thrombotic thrombocytopenic purpura (cTTP) compared with standard therapy.

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