We asked, and you answered! Here are the responses from this month’s “You Make the Call” question on treatment options for a young female patient with beta thalassemia major and pancytopenia who requires frequent blood transfusions.
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My plan of management is as follows:
- Splenectomy is appropriate and could reduce the need for transfusion.
- Stop luspatercept, as it may increase iron absorption.
- Give parenteral iron chelator to control her iron overload.
Hanan Alwazzan, MD, MBBS, MSc
Al Ahmadi Governorate, Kuwait
This 25-year-old patient with beta thalassemia major, pancytopenia, hepatosplenomegaly, and severe iron overload requires a multifaceted treatment approach. My recommendations are:
- Prioritize iron overload management with a triple chelation regimen, including deferiprone, deferasirox dose optimization, and continuous deferoxamine infusion.
- Assess liver health via an MRI for liver iron concentration and noninvasive fibrosis tests, avoiding biopsy due to low platelets.
- Use Doppler imaging to screen for veno-occlusive disease (VOD) and optimize transfusions to maintain hemoglobin at 10 to 12 g/dL.
I would defer gene therapy due to the patient’s high iron overload and liver dysfunction, which increase VOD risk. After improving iron levels and liver health, gene therapy can be reconsidered.
Akshat Jain, MD, MPH
Redlands, CA
