at San Francisco, California
study started
estimated completion
Principal Investigator
by Tippi C MacKenzie, MD
Photo of Tippi C MacKenzie
Tippi C MacKenzie



This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with Alpha thalassemia.

Official Title

International Prospective Registry of Patients With Alpha Thalassemia


The aim of this registry is to prospectively and retrospectively collect data on patients who are diagnosed with alpha thalassemia major and other alpha thalassemia mutations. Data collected will be used to: 1. Identify patient outcomes of therapies. 2. Improve clinical management of patients with ATM. 3. Improve medical decision making. 4. Improve quality of care.


Alpha-Thalassemia Alpha Thalassemia Major Alpha Thalassemia Minor Thalassemia beta-Thalassemia


You can join if…

  • diagnosis of alpha thalassemia (prenatal or postnatal) with genotype consistent with ATM or BHFS phenotype
  • referred to the University of California, San Francisco Fetal Treatment Center for fetal diagnosis, management and/or evaluation for the ongoing in utero stem cell transplantation clinical trial

You CAN'T join if...

  • none


  • University of California San Francisco accepting new patients
    San Francisco California 94143 United States

Lead Scientist at UCSF

  • Tippi C MacKenzie, MD
    Tippi MacKenzie is a pediatric and fetal surgeon who is focused on developing better ways to diagnose and treat genetic diseases before birth. She leads a translational research lab examining the unique biology between the mother and her fetus, with the idea that pregnancy complications such as preterm labor arise from a breakdown in maternal-fetal tolerance.


accepting new patients
Start Date
Completion Date
University of California, San Francisco
Study Type
Observational [Patient Registry]
Last Updated