Past Events

Advances in Alpha Thalassemia Major: No Longer a Fatal Disease

Aired on 1.09.2021
FREE
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 


 

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

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.

 


Speakers

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

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

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

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

Wade Kyono, MD
Kapiolani Medical Center for Women & Children

Barbara Koenig, PhD
University of California, San Francisco

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

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

Craig Butler
Cooley's Anemia Foundation, National Executive Director

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

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

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, University of California, San Francisco

John Waye, MD
Faculty of Health Sciences McMaster University

Mara Rosner, MD, MPH
Johns Hopkins Medicine

Agenda

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

 (All times are Pacific Standard Time)

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
Moderator: 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
Moderator: 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
Introductions: 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

 

(All times are Pacific Standard Time)

3:00 - 3:50 PM

Forum A: Specialists discuss IUT protocol consensus
Moderator: Juan González Velez, MD, PhD (UCSF)

3:00 - 3:50 PM

Forum B: Building a patient-family group for ATM
 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
 Moderator: 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

Supachai Ekwattanakit, MD, PhD (Siriraj Thalassemia Center, Mahidol University)

Alexis Thompson, MD, MPH

Elliott Vichinsky, MD

5:30 PM

Thalassemia International Federation (TIF)

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

Billie Lianoglou, LCGC (UCSF)

Mara Rosner, MD (Johns Hopkins Medicine)

6:25 PM

Close Meeting
Tippi MacKenzie, MD (UCSF)

6:35 PM

ADJOURN

Resources

  

CME & CEU
Up to 7.25 CME units
UCSF Office of Continuing Medical Education
Register
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.