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Blood clot forming | image credit: Tatiana Shepeleva - stock.adobe.com
Prophylaxis With Recombinant ADAMTS13 Demonstrates Superiority vs Standard Therapy in Congenital TTP

June 5th 2024

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.

Blood for Transfusion PNH Concept | image credit: sebgross - stock.adobe.com
Pegcetacoplan Contributes to Better Outcomes in PNH vs Other Treatments

May 29th 2024

Hands Holding Newborn Baby | image credit: BazziBa - stock.adobe.com
SCD Birth Rate Higher in Socially Vulnerable Areas, Study Finds

May 9th 2024

Immune Cells and Antibodies Concept | image credit: vipman4 - stock.adobe.com
Immune Irregularities Detected in Patients With PNH

May 4th 2024

Electronic Health Record Access | image credit: pandpstock001 - stock.adobe.com
EHR Alerts Improve Quality of Care in Heparin-Induced Thrombocytopenia, Study Finds

April 30th 2024

More News

Sickle cell anemia, 3D illustration showing blood vessel with normal and deformed crescent | Image credit: tussik - stock.adobe.com
November 10th 2023

Mitapivat Effective, Safe in Treating SCD Through 1 Year

Julia Bonavitacola
Durable mechanistic effects and a good safety profile were found in patients who took mitapivat for sickle cell disease (SCD).
FDA Approves Adzynma for Treatment of Congenital Thrombotic Thrombocytopenic Purpura
November 10th 2023

FDA Approves Adzynma for Treatment of Congenital Thrombotic Thrombocytopenic Purpura

Julia Bonavitacola
Adzynma is the first product to be approved for the treatment of the rare blood disorder that has 90% mortality when left untreated.
Sickle-cell and normal red blood cells | Image credit: extender_01 - stock.adobe.com
November 7th 2023

Children Hospitalized for Sickle Cell Anemia More Commonly Have AKI

Julia Bonavitacola
Acute kidney injury (AKI) was found to be common in children aged 17 years and younger who were hospitalized for sickle cell anemia.
Pegcetacoplan Found to Be Cost-Effective Approach to Treating PNH
November 4th 2023

Pegcetacoplan Found to Be Cost-Effective Approach to Treating PNH

Julia Bonavitacola
Patients with paroxysmal nocturnal hemoglobinuria (PNH) who had suboptimal response to eculizumab can switch to pegcetacoplan for a cost-effective alternative, according to. acost analysis.
Caplacizumab Effective, Safe in Treating Immune TTP
November 1st 2023

Caplacizumab Effective, Safe in Treating Immune TTP

Julia Bonavitacola
Immune thrombotic thrombocytopenic purpura (TTP) can be treated safely and effectively with caplacizumab.
what we're reading logo
November 1st 2023

What We’re Reading: Panel on Sickle Cell Gene Therapy; Infant Mortality Rises; Campaign Against Burnout

AJMC Staff
A panel of experts agreed that exa-cel, a sickle cell disease gene therapy, was safe enough for clinical use, setting the stage for a potential FDA approval; the United States saw an increase in infant mortality rates for the first time in more than 2 decades; the new antiburnout campaign from the CDC asks leaders to better support health care workers.
What We’re Reading: Pharmacy Staff Stage Nationwide Walkout; Dads Embrace Paid Family Leave; FDA Reviews Therapy for Sickle Cell Disease
October 30th 2023

What We’re Reading: Pharmacy Staff Walkout; Male Parental Leave; FDA Reviewing SCD Therapy

AJMC Staff
Pharmacy workers embark on a multiday strike; Fathers are taking paid family leave to bond with their newborns; the FDA is set to evaluate a pioneering gene therapy for sickle cell disease (SCD).
Eculizumab Shows Long-Term Efficacy, Safety in PNH
October 26th 2023

Eculizumab Shows Long-Term Efficacy, Safety in PNH

Julia Bonavitacola
Paroxysmal nocturnal hemoglobinuria (PNH) could be effectively treated by using eculizumab, which was found to be safe in patients.
Racial, Ethnic Differences Persist in Nurses, Nursing Assistants When Treating Sickle Cell Disease
October 25th 2023

Racial, Ethnic Differences Persist in Nurses, Nursing Assistants When Treating Sickle Cell Disease

Julia Bonavitacola
Biases in care for young adults with sickle cell disease could lead to lower quality of care for these patients.
Non-ADAMTS13 Parameters Could Benefit Differential Diagnosis in TTP
October 20th 2023

Non-ADAMTS13 Parameters Could Benefit Differential Diagnosis in TTP

Julia Bonavitacola
Including biomarkers for non-ADAMTS13 in routine clinical testing could benefit diagnosis and follow-up in patients with immune-mediated thrombotic thrombocytopenic purpura (TTP).
Heavy Menstrual Bleeding, Dysmenorrhea High in Adolescents With Sickle Cell Disease
October 17th 2023

Heavy Menstrual Bleeding, Dysmenorrhea High in Adolescents With Sickle Cell Disease

Julia Bonavitacola
Young women with sickle cell disease have a higher prevalence of heavy menstrual bleeding and dysmenorrhea.
Lactate Dehydrogenase Levels Associated With Fatigue, QOL in Patients With PNH
October 13th 2023

Lactate Dehydrogenase Levels Associated With Fatigue, QOL in Patients With PNH

Julia Bonavitacola
Patients with paroxysmal nocturnal hemoglobinuria (PNH) had decreased quality of life (QOL) and worse fatigue that could be linked to lactate dehydrogenase levels.
Platelet Count Acted as Predictor of Relapse in TTP
October 11th 2023

Platelet Count Acted as Predictor of Relapse in TTP

Julia Bonavitacola
The 3-year relapse and mortality rates for thrombotic thrombocytopenic purpura (TTP) were 23.6% and 26.3%, predicated on platelet count.
Children With SCD Have Limited Gastrointestinal Evaluations
October 6th 2023

Children With SCD Have Limited Gastrointestinal Evaluations

Julia Bonavitacola
Although children with sickle cell disease (SCD) frequently present with abdominal pain, limited gastrointestinal evaluations are performed for them.
Sickle Cell Disease Associated With Severe COVID-19, Mortality
October 3rd 2023

Sickle Cell Disease Associated With Severe COVID-19, Mortality

Julia Bonavitacola
Severe cases of COVID-19 and COVID-19–related deaths were associated with sickle cell disease.
Adverse Effects of Hospitalization for Vaso-Occlusive Crises Persist for 12 Months
September 28th 2023

Adverse Effects of Hospitalization for Vaso-Occlusive Crises Persist for 12 Months

Julia Bonavitacola
Children with sickle cell disease who require hospitalization for vaso-occlusive crises can experience adverse effects for up to 12 months following hospitalization, a study found.
Doctor talking to patient
September 22nd 2023

Patient-Reported Outcome Measures Pick Up Patient Experiences in Patients With TTP

Julia Bonavitacola
Patients with thrombotic thrombocytopenic purpura (TTP) benefited from patient-reported outcome measures to report their quality of life.
Relative Benefit in Survival, Reduction in TE/MAVE Risk With Eculizumab for Patients With PNH
September 19th 2023

Relative Benefit in Survival, Reduction in TE/MAVE Risk With Eculizumab for Patients With PNH

Julia Bonavitacola
When patients with paroxysmal nocturnal hemoglobinuria (PNH) received eculizumab, survival rates increased and thromboembolic events/major adverse vascular events (TE/MAVE) decreased.
Study Identifies Risk Factors for Thromboembolic Events in Patients With PNH
September 14th 2023

Study Identifies Risk Factors for Thromboembolic Events in Patients With PNH

Julia Bonavitacola
Researchers found indicators that increased the risk of thromboembolic events in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Repeated Molecular Testing Indicates Dynamic Disease Profile in Patients With PV, ET
September 12th 2023

Repeated Molecular Testing Indicates Dynamic Disease Profile in Patients With PV, ET

Julia Bonavitacola
Patients with polycythemia vera (PV) and essential thrombocythemia (ET) require repeated molecular testing and improved prognostic tools.
Processing Speed May Be Slower in People Who Have SCD
September 8th 2023

Processing Speed May Be Slower in People Who Have SCD

Julia Bonavitacola
Patients who have with sickle cell disease (SCD) could have a slower processing speed and higher cerebral blood flow compared with controls.
Blood cells
September 5th 2023

Imatinib Could Be Therapeutic Option for Treating Painful Sickle Cell Crises

Julia Bonavitacola
Painful crises in sickle cell disease or sickle cell anemia could potentially be treated using imatinib as a therapeutic option, a small study found.
Group of doctors
August 28th 2023

Study Finds Simulation Useful to Boost Awareness of Osteomyelitis in SCD Among Residents, Fellows

Julia Bonavitacola
A simulation case for emergency medicine residents was found to be helpful as a review of the medical diagnosis and management of osteomyelitis in patients with pediatric sickle cell disease (SCD).
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).
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