Past Events

Advances in Alpha Thalassemia Major: No Longer a Fatal Disease

Aired on 1.09.2021
Advances in Alpha Thalassemia Major: No Longer a Fatal Disease CME

January 8, 2021, 4:00 - 7:30 pm PST

January 9, 2021, 3:00 - 6:30 pm PST

Pacific Standard Time/Date
Check the date and time where you live 




Alpha Thalassemia Major (ATM) was once considered universally fatal in utero.  If left untreated, a fetus with ATM will develop severe anemia and hydrops and will die before or shortly after birth. However, recent evidence from patient registries has demonstrated that when ATM is diagnosed early in pregnancy, in utero transfusions (IUT) can reverse hydrops and enable survival with good neurological outcomes. Still, widespread adoption of IUTs - commonly administered to treat other fetal conditions - has not materialized for patients with ATM. 

Conference goals:

    • Demonstrate the impact of prenatal therapy on postnatal and long-term outcomes of Alpha Thalassemia Major (ATM)
    • Understand best practices for prenatal and postnatal treatment of ATM
    • Bring together families affected by ATM to share their diagnosis and treatment journeys

In collaboration with the Cooley’s Anemia Foundation and the Thalassaemia International Federation (TIF), we will hold a special forum dedicated to building a patient group for ATM.

 Two half-day sessions are organized around (Day 1) prenatal screening, diagnosis, and therapies for ATM pregnancies, and (Day 2) postnatal management of ATM from birth to adulthood. Participants include clinicians, scientists, and families affected by ATM.

Prenatal topics include the natural history of ATM, ultrasound diagnosis of hydrops fetalis, genetic diagnosis of ATM, management of pregnancies affected by ATM, protocols for IUT, and an update on the ongoing phase 1 clinical trial of in utero hematopoietic stem cell transplant at the University of California, San Francisco.  We will hold a specific forum to outline best practices for prenatal management with IUT.

Postnatal topics include protocols for chronic transfusions, bone marrow transplantation, and complications of ATM management. Barriers that inhibit access to prenatal and postnatal treatment, and strategies to make therapies more accessible globally will be discussed.

Note regarding schedule: Because ATM disproportionately affects individuals of Southeast Asian ancestry, we planned the conference so that people living in Southeast Asia and Pacific Islands can participate during daytime hours. All are welcome.

Sponsored by the Center for Maternal-Fetal Precision Medicine at the University of California, San Francisco, and the California Institute for Regenerative Medicine.




Mara Rosner, MD, MPH

John Waye, MD

Vip Viprakasit, MD, DPhil

Elliott Vichinsky, MD

Alexis Thompson, MD, MPH

Marisa Schwab, MD

Greg Ryan, MB

Pornpimol Ruangvutilert, MD, PhD

Tachjaree Panchalee, MD

Keith Ogasawara, MD

Mary Norton, MD

Ali Amid, MD

Craig Butler

Sandy Gilbert, PhD

Juan Gonzáles Velez, MD, PhD

Barbara Koenig, PhD

Wade Kyono, MD

Ashutosh Lal, MD

Billie Lianoglou, LCGC

Roberta Keller, MD


Topics Covered



UCSF Fetal Treatment Center | Alpha Thalassemia Major

Prenatal Screening & Diagnosis Algorithm for Thalassemia

NYT: Five Blood Transfusions, One Bone Marrow Transplant -- All Before Birth

VIDEO: Overview of Alpha Thalassemia Major, in utero transfusions, and the UCSF clinical trial of In Utero Stem Cell Transplantation



Up to 7.25 CME units

UCSF Office of Continuing Medical Education


The University of California, San Francisco (UCSF) is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians and allied health professionals.


Up to 0.554 Category 1 CEU

National Society for Genetic Counselors  

The National Society for Genetic Counselors (NSGC) is accredited by the American Board of Genetic Counseling (ABGC) to provide Continuing Education Units (CEUs) for genetic counselor certification and recertification. NSGC has authorized the University of California San Francisco to offer up to 0.554 CEUs or 5.54 Category 1 contact hours for the activity Alpha Thalassemia Major: No Longer a Fatal Disease, which will be accepted by ABGC for genetic counselor certification and recertification.

Process to obtain NSGC CEU credit

Register and attend the conference.

After the conference:

·      You will be asked to complete an evaluation on which you will include your NSGC user id.  (If you do not have a user ID, you can register for a guest id here.) 

·      Pay $25 CEU fee (instructions pending)

·      Meeting staff will confirm your contact hours, then send your NSGC user id, evaluation, and payment to NSGC. NSCG will issue the certificate.