Past Events

Advances in Alpha Thalassemia Major: No Longer a Fatal Disease

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

UCSF hosted an international conference on alpha thalassemia major (ATM) for healthcare providers and families. Part 1: Prenatal screening, diagnosis, and therapies for ATM pregnancies. Part 2: Postnatal management of ATM from birth to adulthood. Short talks and panel discussions with clinician-scientists, and conversations with families about their prenatal diagnosis and treatment journeys.

Description

CONFERENCE FOR PATIENTS AND PROVIDERS

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
    • Build international connections among scientists, medical professionals, and
      patient advocates to advance treatments for ATM worldwide

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. Day 2 opens with a specialists’ 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 are 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.

 

Partners include the UCSF Fetal Treatment Center, the Northern California Comprehensive Thalassemia Center, the Cooley’s Anemia Foundation, and the Thalassaemia International Federation.


 


Speakers

Tippi MacKenzie, MD, Conference Chair
UCSF Benioff Children’s Hospital, University of California, San Francisco

Ali Amid, MD
Children's Hospital of Eastern Ontario, University of Ottawa

Craig Butler
Cooley's Anemia Foundation, National Executive Director

Sandy Gilbert, PhD
Cooley's Anemia Foundation, Senior Director of Medical Affairs

Juan Gonzáles Velez, MD, PhD
UCSF Benioff Children’s Hospital, University of California, San Francisco

Roberta Keller, MD
UCSF Benioff Children’s Hospital, University of California, San Francisco

Barbara Koenig, PhD
University of California, San Francisco

Wade Kyono, MD
Kapiolani Medical Center for Women & Children

Ashutosh Lal, MD
UCSF Benioff Children’s Hospital, University of California, San Francisco

Billie Lianoglou, LCGC
UCSF Benioff Children’s Hospital, University of California, San Francisco

Mary Norton, MD
UCSF Benioff Children’s Hospital, University of California, San Francisco

Keith Ogasawara, MD
Kaiser Permanente Hawaii

Tachjaree Panchalee, MD
Faculty of Medicine Siriraj Hospital, Mahidol University, University of California, San Francisco

Mara Rosner, MD, MPH
Johns Hopkins Medicine

Pornpimol Ruangvutilert, MD, PhD
Faculty of Medicine Siriraj Hospital, Mahidol University

Greg Ryan, MB
Ontario Fetal Centre, University of Toronto

Marisa Schwab, MD
UCSF Benioff Children’s Hospital, University of California, San Francisco

Alexis Thompson, MD, MPH
Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine

Elliott Vichinsky, MD
UCSF Benioff Children’s Hospital, University of California, San Francisco

Vip Viprakasit, MD, DPhil
Faculty of Medicine Siriraj Hospital, Mahidol University

John Waye, MD
Faculty of Health Sciences, McMaster University

Agenda

DAY 1, January 8, 2021  Pacific Standard Time/Date

4:00 PM

Surviving with Joy – a short film

4:10 PM

Welcome and overview – Day 1
Tippi MacKenzie, MD (UCSF)

4:20 PM

Family Experience 1
Conversations with families about their diagnosis and treatment journeys

4:50 PM

Lessons from Patient Registries
Introduction: Ali Amid, MD


Canada Registry of ATM outcomes 
Ali Amid, MD (Children's Hospital of Eastern Ontario, University of Ottawa)

Hong Kong Registry of ATM outcomes 
Wilson YK Chan, MBBS (Queen Mary Hospital, Hong Kong University)


UCSF Registry – focus on neurological outcomes
Marisa Schwab, MD, Billie Lianoglou, LCGC (UCSF) 


Meta-analysis of aggregated registry data
Tachjaree Panchalee, MD (Siriraj Hospital, Mahidol University) 


Live Q&A

5:55 PM

BREAK 

6:00 PM 

Prenatal Screening and Diagnosis
Introduction: Mary Norton, MD


Genetic diagnosis of ATM
John Waye, PhD (McMaster University)  


Prenatal thalassemia screening algorithm
Mary Norton, MD (UCSF)


Ultrasound diagnosis of hydrops
Pornpimol Ruangvutilert, MD, PhD (Siriraj Hospital, Mahidol University)


Live Q&A

6:45 PM

BREAK

6:50 PM

Optimizing fetal therapy for ATM
Introduction: Keith Ogasawara, MD


Managing pregnancies affected by ATM 
Juan González Velez, MD, PhD (UCSF)


Adoption of IUT as a therapy for ATM in the State of Hawaii
Keith Ogasawara, MD (Kaiser Permanente Hawaii)


In utero transfusion protocol
Greg Ryan, MB (Ontario Fetal Centre, University of Toronto)


In utero hematopoietic stem cell transplantation - Phase 1 clinical trial
Tippi MacKenzie, MD (UCSF)

7:20 PM

Live panel: Interdisciplinary discussion regarding prenatal care for ATM
Moderator: Tippi MacKenzie, MD

Juan González Velez, MD, PhD
Roberta Keller, MD (UCSF)
Keith Ogasawara, MD
Greg Ryan, MB

7:50 PM

Close Meeting
Tippi MacKenzie, MD

8:00 PM

ADJOURN

 

DAY 2, January 9, 2021  Pacific Standard Time/Date 

3:00 - 3:50 PM

Forum A: Specialists discuss IUT protocol consensus
Moderators: Juan González Velez, MD, PhD, Billie Lianoglou, LCGC (UCSF)

3:00 - 3:50 PM

Forum B: Building a patient-family group for ATM    (not recorded)
Moderator: Sandra Gilbert, PhD (Cooley's Anemia Foundation)

4:00 PM

Welcome and overview - Day 2
Tippi MacKenzie, MD (UCSF)

4:05 PM

Family Experience 2
Conversations with families living with ATM

4:35 PM

Postnatal therapies for ATM
Introduction: Vip Viprakasit, MD, DPhil


Postnatal transfusion protocols
Elliott Vichinsky, MD (UCSF)


Postnatal hematopoietic stem cell transplantation
Alexis Thompson, MD, MPH (Lurie Children's Hospital of Chicago, Northwestern Feinberg School of Medicine) 


Transfusion and transplantation in Thailand
Vip Viprakasit, MD, PhD (Siriraj Hospital, Mahidol University)

5:05 PM

Live panel: Interdisciplinary discussion regarding opportunities and challenges to providing postnatal care for ATM
Moderator: Vip Viprakasit, MD, DPhil

Kay Thwe Aung, MD (Mandalay Children's Hospital)
Maria Cecilia Cruz , MD (Philippine Children's Medical Center)
Supachai Ekwattanakit, MD, PhD (Siriraj Thalassemia Center, Mahidol University)
Sandhya Kharbanda, MD (UCSF)
Aye Aye Khaing, MD (Yangon Children′s Hospital)
Alexis Thompson, MD, MPH
Elliott Vichinsky, MD

5:30 PM

Thalassaemia International Federation (TIF)
Michael Angastiniotis, MBChB, TIF Medical Advisor 

5:35 PM

BREAK

5:40 PM

Live Panel: Discussion with families and providers
Introduction by Craig Butler, Executive Director, Cooley's Anemia Foundation
Moderator: Barbara Koenig, PhD, Director, UCSF Bioethics Program
Melanie Kirby-Allen, MD (Hospital for Sick Children)
Billie Lianoglou, LCGC (UCSF)
Mara Rosner, MD (Johns Hopkins Medicine)
Family representatives

6:25 PM

Close Meeting
Tippi MacKenzie, MD (UCSF)

6:35 PM

ADJOURN

 

Prenatal Care for Alpha Thalassemia Major (ATM) – Resources from UCSF Fetal Treatment Center

English (scroll down for other languages)


 

Non-English Resources

Whiteboard Animation - Fetal Therapy for ATM (video)
Thai – CantoneseMandarinVietnameseTagalog
 
Provider Brochure − Fetal Intervention for ATM
Thai Traditional ChineseVietnameseTagalog