Murdoch Children’s Research Institute’s COVID Vaccine Preparedness survey

COVID-19 vaccine delivery may be prioritised for people aged 70 years-old and older, and for adults with chronic conditions like heart disease, respiratory conditions, diabetes, neurological conditions, immunocompromising conditions, renal disease or haematological disorders.

If you’re in one of these groups, we want to hear from you!

What do you want to know about the COVID-19 vaccines, and how do you want to get that information?

Never before have we undertaken a vaccine program on this scale. We want to understand what is needed to help you make decisions about getting vaccinated. Help shape the Victorian COVID-19 vaccination communication strategy by completing the Murdoch Children’s Research Institute’s COVID Vaccine Preparedness survey:

https://redcap.link/COVID19Vaccine

This online survey takes 10 minutes and can be completed anonymously. Participants can enter to win one of eight gift vouchers.

This study is led by researchers from the Murdoch Children’s Research Institute with partners from the University of Melbourne, Monash University, The University of Sydney and the University of New South Wales. It is funded by the Victorian Department of Health.

To learn more about the study and complete the survey, click HERE

Please share this survey with family and friends who may be eligible. If you have any questions, please contact Dr Jessica Kaufman 03 9345 4890 or COVIDVaccine@mcri.edu.au

Bluebird Bio Announces Temporary Suspension on clinical trials of LentiGlobin Gene Therapy for Sickle Cell Disease

Bluebird Bio, developer of gene therapy Zynteglo, has suspended their clinical study of the treatment on sickle cell patients after adverse events were reported. Two patients developed Acute Myeloid Leukemia (AML), a type of blood and bone marrow cancer, after being treated with Zynteglo five years ago.

A patient was reported last week developing Myelodysplastic Syndrome (MDS), a blood cell production disorder, This follows a case of MDS in 2018 that was attributed to the chemotherapy received in preparation for gene therapy.

It is currently unknown as to whether the development of AML was caused by the viral vector used in the therapy. Further investigations are underway.

No cases of hematologic malignancy such as AML or MDS has been reported in the use of Zynteglo for β-thalassaemia so far. However, the marketing of Zynteglo in Europe has been suspended as a precautionary measure.

Zynteglo, previously known as Lentiglobin, is a gene therapy developed by US Biotechnology company Bluebird Bio for the treatment of sickle cell disease and transfusion-dependent β-thalassaemia. The therapy uses a viral vector to deliver genetic changes to patients with the hope of improving haemoglobin and red blood cell production.

European Medicines Agency Accepts GBT’s Marketing Authorization Application (MAA) for Oxbryta® (voxelotor) for the Treatment of Hemolytic Anemia in Sickle Cell Disease

Global Blood Therapeutics, Inc. (GBT)  announced that the European Medicines Agency (EMA) has completed the validation of GBT’s Marketing Authorization Application (MAA) for Oxbryta (voxelotor) tablets and started its standard review process.

A first-in-class oral, once-daily therapy, Oxbryta directly inhibits haemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. The sickling process causes hemolytic anaemia (low haemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.

“Sickle cell disease has a devastating impact on the lives of patients and their families, including serious and life-threatening complications that can lead to organ damage and early death,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Despite this overwhelming need, there are currently no approved therapies in Europe that have the potential to modify the course of the disease. We look forward to working with the EMA to meet our goal of bringing the first treatment for hemolytic anaemia in sickle cell disease to European patients as soon as possible.”

The marketing authorisation application is based on data from the Phase III HOPE study and the Phase II HOPE-KIDS 1 study, both of which enrolled patients at clinical sites in Europe. The HOPE study achieved its primary endpoint.

The analysis of the complete data from the HOPE study further demonstrated that Oxbryta, at a daily dose of 1,500mg, resulted in durable improvements in haemoglobin levels and markers of haemolysis over 72 weeks of treatment.

Oxbryta has already been approved in the US for the treatment of SCD in adults and children 12 years of age and older.

Read the Full GBT Press Release HERE

Haemoglobinopathy Registry at Monash Health

Monash Health is pleased to be participating in the Haemoglobinopathy Registry (HbR), a national database set up to monitor the health of Australians living with thalassaemia, sickle cell disease and other disorders of haemoglobin, and the medical care they receive.  

 

The registry is an important first step in a major project, which aims to ensure that all Australians living with a haemoglobinopathy receive the best possible medical care, now and in the years to come.  The more people who participate in the registry, the better researchers will be able to assess the current situation and help plan for the future. 

 

The Haemoglobinopathy Registry is managed by Monash University and has been approved by the Monash Health Research Ethics Committee. We have enclosed a registry information brochure, which describes what your participation means and how your privacy is protected.  Participation is voluntary and will have no impact on the care you receive. 

 

This registry is an ‘opt‐out’ registry. This means that unless you inform us within 14 days of the date of this letter that you do not wish to participate, we will begin entering your details into the database.  But even if you miss this cut‐off, you can still withdraw from the registry at any time, by calling the HbR project officer on freecall 1800 811 326, emailing hbr@monash.edu, or sending in the ‘Decline to Participate’ slip attached to the information brochure.

Do you have β-thalassaemia? We want to hear from you!

Monash University is currently conducting a study to better understand what men and women with beta thalassaemia know and think about fertility and pregnancy. We want to use this information to design educational materials to help people with thalassaemia better understand how thalassaemia affects fertility and how to achieve a safe and healthy pregnancy! 

All we need you to do is fill out an online survey. It will only take 20 minutes to complete and will ask you about your experiences and knowledge about contraception, fertility and pregnancy in thalassaemia. The survey responses are completely confidential and anonymous.

To access the survey, please click here. You can complete the survey anytime on your computer, phone or any other device!

The more people who complete the survey, the better we can make the educational materials, so if you know anyone who would be interested in participating please pass the link on!