Hematologic Disorders

Many of the more “common” blood diseases are considered “rare,” affecting less than 200,000 U.S. citizens. These conditions primarily affect the blood & blood-forming organs. In this section we provide news, research summaries, and meeting reports relevant to the hematologic community.
Feb 14, 2025| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment

Results from the GLOW Clinical Trial in Chronic Lymphocytic Leukemia

Carsten Utoft Niemann, MD, PhD, Copenhagen University Hospital,…
Feb 12, 2025| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment

Results from the MajesTEC-2 and TRIMM-2 Clinical Trials for Patients With Multiple Myeloma

Anita D’Souza, MD, Medical College of Wisconsin, discusses…
Feb 11, 2025| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment

Phase 2 REVIVE Clinical Trial for Polycythemia Vera

Arturo Molina, MD, Protagonist Therapeutics, discusses results from…
Feb 5, 2025| Posted in: Cancers, Hematologic Disorders, Treatment

Chemotherapy Combination Treatment in Patients with Acute Myeloid Leukemia

Emma Searle, PhD, The Christie NHS Foundation Trust…
Feb 4, 2025| Posted in: Advocacy, Cancers, Hematologic Disorders, Treatment

Differences in Clinical Outcomes of Black Patients With Multiple Myeloma

Carolina Schinke, MD, University of Arkansas for Medical…
Feb 3, 2025| Posted in: Drug Development, Hematologic Disorders, Treatment

Combination C5 Therapy Versus Standard of Care in Paroxysmal Nocturnal Hemoglobinuria

Christopher Patriquin, MD, University of Toronto, discusses results…
Jan 30, 2025| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment

GMMG-HD7 Clinical Trial for Patients With Multiple Myeloma

Elias Mai, MD, Heidelberg Myeloma Center, discusses the…
Jan 28, 2025| Posted in: Cancers, Drug Development, Hematologic Disorders, Treatment

Results From the PERSEUS Clinical Trial in Multiple Myeloma

Pieter Sonneveld, MD, Erasmus MC Cancer Institute, discusses…

Recent Videos

Hematologic Malignancies and Clinical Trial Participation: A Shared Decision-Making Approach

This 30-minute, CME-accredited program, hosted by John Kuruvilla, MD, explores best practices for discussing possible clinical trial participation with patients who have hematologic malignancies.

Overview of Epigenetics and Epigenomics in Lysosomal Disorders

Lysosomal storage diseases are a group of approximately 50 rare inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.

Rates of Severe Neutropenia and Infection Risk in Patients Treated With Deferiprone: 28 Years of Data

Dr. Sujit Sheth, Chief of the Division of Pediatric Hematology/Oncology at Weill Cornell Medicine and an attending pediatrician at New York Presbyterian Hospital, in New York City discusses his recently published work in Blood Advances titled “Rates of severe neutropenia and infection risk in patients treated with deferiprone: 28 years of data.”

Cutaneous T-Cell Lymphoma

Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders.[1] Cutaneous T-cell lymphoma is a rare group of malignancies, with an incidence of 6.4 cases per 1 million people. This form of T-cell lymphoma represents around 70% of primary cutaneous lymphomas.

Epigenetic and Epigenomics Signature in Lysosomal Disorders Pathology

Lysosomal storage diseases are a group of approximately 50 rare inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.

Understanding the Global Differences in Lysosomal Disorders for Patient Care

Lysosomal storage diseases are a group of approximately 50 rare inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies.

Hematologic Disorders

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Carsten Utoft Niemann, MD, PhD, Copenhagen University Hospital, discusses results from the GLOW clinical trial in chronic lymphocytic leukemia (CLL).

https://checkrare.com/results-from-the-glow-clinical-trial-in-chronic-lymphocytic-leukemia/

#CheckRare #RareDisease #RareCancer

📢CME Program: Jean Elwing, MD, of the University of Cincinnati College of Medicine provides an overview of the latest clinical research about PAH presented at CHEST 2024.

https://checkrare.com/learning/p-pah-clinical-research-highlights-chest-2024/

#CheckRare #RareDisease ...#RareLung #CMEProgram

FDA Approval: The U.S. Food and Drug Administration has approved Gomekli (mirdametinib) for the treatment of adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.

https://checkrare.com/fda-approves-mirdametinib-for-treatment-of-nf1-pn/

...#CheckRare #FDAApproval #RareGenetic

💡Rare Disease Spotlight: Polycythemia Vera

Learn more about this rare disease at https://checkrare.com/?s=polycythemia+vera

#CheckRare #RareDisease #PolycythemiaVera #RareHematology

Clinical research leaders in HAE provide an overview of the discrepancies in care observed in certain patient populations, as well as a discussion on best practices to reduce those inequalities moving forward.

...https://checkrare.com/improving-health-equity-in-hereditary-angioedema-hae-a-panel-discussion/

#CheckRare #RareDisease #RareGenetic

📹 The CheckRare team spent the week at #WORLDSymposia 2025

Stay tuned for our interviews from the conference!

#CheckRare #RareDisease

Paula Ragan, President and Chief Executive Officer of X4 Pharmaceuticals, discusses the pathway of approval under the European Medicines Agency (EMA) for mavorixafor for patients with WHIM syndrome.

...https://checkrare.com/ema-pathway-to-approval-for-mavorixafor-to-treat-patients-with-whim-syndrome/

#CheckRare #RareDisease #WHIMSyndrome #RareGenetic #RareSkin

Emma Searle, PhD, The Christie NHS Foundation Trust and University of Manchester, discusses bleximenib in combination with chemotherapy to treat patients with acute myeloid leukemia (AML).

https://checkrare.com/chemotherapy-combination-treatment-in-patients-with-acute-myeloid-leukemia/
...
#CheckRare #RareDisease #AML #RareCancer

February 4th is #WorldCancerDay

Although there are over 200 types of rare cancers, only a small number of people are diagnosed with each type. These conditions often present unique challenges in diagnosis, research, and treatment. Today we raise awareness, support research, and advocate ...for better resources.🎗️

To learn more about rare cancers, visit https://checkrare.com/diseases/cancers/

#CheckRare #RareDisease #RareCancer

Caplacizumab Combination Therapy in Patients With Acquired Thrombotic Thrombocytopenic Purpura

Jean Donadieu, MD, Service d’Hémato-Oncologie Pédiatrique, Hopital, Paris, provides an overview of WHIM syndrome.

https://checkrare.com/whim-syndrome-overview-diagnosis-and-magement/

#CheckRare #RareDisease #RareGenetic #RareSkin #WHIM

Join Professors João Gonçalves and Paolo Calicaeti in this educational webinar discussing how PEGylation represents a major technological advancement in the development and optimization of therapeutic proteins.

...https://checkrare.com/optimizing-therapeutic-proteins-through-pegylation/

#CheckRare #RareDisease #lysosomal #RareLysosomal