Genetic Blood Disorders

Genetic Blood Disorders Content

New hope for sickle cell treatment

Researchers at Vanderbilt University Medical Center (VUMC) have highlighted promising results from a phase II clinical trial for a new treatment for sickle cell disease (SCD). Published in Blood, the study aimed to achieve an 80% two-year event-free survival rate following a specific bone marrow transplant (BMT) method. The international trial, led by Dr DeBaun and Dr Adetola Kassim, involved 32 children and 38 adults from five countries.

Paediatric Haematology, Haematological Malignancies, Genetic Blood Disorders

Watch Time: 48 mins

touchMDT

Recognizing and managing HLH in the critical care setting: Best practice for the multidisciplinary team

CE/CME ACCREDITED Prof. Michael Jordan, Dr Kate Kernan, Dr Eric Gars, Prof. Rob MacLaren Translations available:

Multidisciplinary experts discuss the diagnosis and management of haemophagocytic lymphohistiocytosis (HLH)

Learning objectives

Learning Objectives

Describe the characteristic clinical and laboratory findings of HLH, and the confirmatory diagnostic tests
Explain the identification of predisposing conditions and acute triggers associated with HLH and how these affect treatment selection and monitoring
Implement effective multidisciplinary strategies for the diagnosis, monitoring and management of HLH in the critical care setting

Genetic Blood Disorders

Watch Time: 39 mins

touchPANEL DISCUSSION

Contemporary insights on the management of sickle cell disease: Focus on complications and recent advances in therapy

CE/CME ACCREDITED Prof. Biree Andemariam, Prof. Modupe Idowu, Prof. Mark C Walters

Three experts discuss the management of complications of sickle cell disease, as well as recent advances in therapy.

Learning objectives

Learning Objectives

Summarize the various manifestations and multiple complications of SCD
Appraise the practical considerations for the multidisciplinary management of SCD complications
Interpret the clinical data for established and recently approved therapies for SCD and apply them to clinical practice

Genetic Blood Disorders

Watch Time: 37 mins

touchPANEL DISCUSSION

Navigating pyruvate kinase deficiency today: How can the disease burden and unmet treatment needs be addressed?

CE/CME ACCREDITED Dr Rachael Grace, Dr Hanny Al-Samkari, Dr Ami J Shah

Three experts discuss the burden and clinical presentation of pyruvate kinase deficiency, as well as current and emerging therapeutic strategies.

Learning objectives

Learning Objectives

Recognize the signs and symptoms of PK deficiency and the burden of disease in affected patients
Evaluate established therapeutic strategies for PK deficiency and identify remaining unmet treatment needs
Describe novel and emerging therapeutic strategies for PK deficiency and how they may address current unmet needs

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Genetic Blood Disorders