International Alpha Thalassemia Consortium

The International Alpha Thalassemia Consortium, hosted by the UCSF Center for Maternal-Fetal Precision Medicine, brings together an interdisciplinary group of clinicians and scientists from around the world to focus expertise in hematology, maternal-fetal medicine, genetics, fetal therapy, and neonatology on alpha thalassemia major (ATM).

We gather quarterly in a virtual seminar to share advances, challenges, and best practices regarding clinical care for prenatal, pediatric, and adult patients diagnosed with ATM.

All are welcome.

Next Quarterly Seminar


Alpha Thalassemia Patient Registry & Clinical Trial

To refer a patient to the patient registry or clinical trial, please contact


The aim of this registry is to prospectively and retrospectively collect data on patients who are diagnosed with alpha thalassemia major (ATM) and other alpha thalassemia mutations. Data collected will be used to:

  1. Understand the natural history of the disease
  2. Identify patient outcomes of therapies
  3. Improve clinical management of patients with ATM
  4. Improve medical decision making
  5. Improve quality of care


Clinical trial to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with ATM, performed at the time of in utero transfusion of red blood cells.

Learn more about this trial at the UCSF Fetal Treatment Center.



Past Seminars

January 23, 2024

Southeast Asian Ovalocytosis: An Alpha Thalassemia Major Imposter?


  • Prenatal diagnosis of fetal anemia
  • Epidemiology and typical presentation of homozygous Southeast Asian Ovalocytosis (compared to Alpha Thalassemia Major (ATM))
  • Prenatal diagnosis of Southeast Asian Ovalocytosis (compared to ATM)
  • Prognosis in untreated patients (compared to ATM) based on literature and our case series
  • Conditions associated with Southeast Asian Ovalocytosis


November 7, 2023

A recording is not available.

  • An overview of gene therapy
  • Beta globin lentiviral vectors developed for sickle cell disease
  • Development of alpha globin lentiviral vectors for alpha thalassemia major (ATM) based on a clinical beta globin vectors
  • Assessment of candidate lentiviral vectors in healthy donor and ATM patient hematopoietic stem and progenitor cells derived into red blood cells


August 15, 2023


  • Ethnic distribution in Singapore and magnitude of problem with Bart’s Hydrops Fetalis Syndrome (BHFS)
  • Conventional practice for BHFS in Singapore
  • Case presentations and lessons learnt
  • Evolution of management including Intrauterine Transfusion (IUT)
  • Future management strategy for BHFS in Singapore


May 4, 2023


  • Current results of unrelated donor hematopoietic stem cell transplantation (URD HCT) for thalassemia and key determinants that determine outcome
  • URD HCT vs gene therapy for transfusion dependent thalassemias (TDT) with regard to accessibility and other considerations
  • Key curative endpoints in curative therapies for TDT


February 15, 2023


  • Review of chelators
  • Challenges in dosing infants
  • Model based dosing


November 15, 2022


Hypospadias is a condition that often develops in male fetuses with alpha thalassemia major.

  • Standard versus severe hypospadias
  • Embryology
  • Etiology
  • Treatment
  • Outcomes


August 23, 2022


  • Three-pronged approach to develop gene therapies to cure alpha thalassemia major ex vivo
  • In-depth look at CRISPR/AAV6-mediated genome editing correction strategy
  • Future directions that include the potential adaptation of successful ex vivo strategies for in utero and postnatal in vivo delivery


May 9, 2022





February 22, 2022


  • Patients registry demographics
  • Outcomes in patients that pursued fetal therapy
  • Silver linings: unintended outcomes from referring provider collaborations


November 30, 2021


  • Risk of iron overload and its related complications in alpha thalassemia patients
  • Standard of care practices for iron overload monitoring
  • Review the currently available options for iron chelation


August 17, 2021


  • How and when to initiate invasive diagnostics and transfusion therapy in fetuses suspected to have alpha thalassemia
  • Technical aspects of determining how much to transfuse, the frequency of transfusions, and the role and technical considerations for fetal exchange transfusion
  • Troubleshooting strategies for difficult cases such as fetuses requiring transfusion at early gestational ages


May 20, 2021


  • current practices in hematopoietic stem cell transplantation for ATM, and upcoming advances that improve survival of babies with ATM


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