ISTH Gene Therapy in Hemophilia Train-the-Trainer


The ISTH is ready to help the hemophilia community prepare for the future approval of a gene therapy in hemophilia. Education and tools have been created to assist you (the trainer) and your team on the path to preparedness. Participate in the ISTH Gene Therapy in Hemophilia Train-the-Trainer program by following the steps below:

  1. Complete the hemophilia baseline survey (optional)
  2. Attend a live virtual training session, or review the enduring module if unable to participate in real time
  3. Complete the quick post assessment survey, which will measure readiness to disseminate education and training to your community
  4. If the survey indicates the need for more education, modules will be suggested for review
  5. Access the educational training tools
  6. Work by yourself or with your hemophilia treatment center (HTC) care team to complete the Change Guides
  7. Stay in touch with peers and experts from around the world by engaging in the discussion boards
STEP 1: Gene Therapy in Hemophilia Baseline Survey – Determine Needs

This survey is an optional tool to identify you or your team’s educational needs. Trainers are encouraged to complete the survey before attending one of the live virtual sessions, or if training others they should ask their teams to complete it. Responses will help tailor content to their specific needs. It can also be used before and after a team training session to serve as a baseline and follow-up assessment for advances in knowledge and confidence.

Step 2: Train-the-Trainer Session – Complete Training

Watch the training session. After this session, you will have access to educational support tools via discussion boards.

Let us know how you are doing. Are there topics for which you need additional education, or further help to support your training? It is important to us that you understand the content well enough to be able to educate others.

Step 3: Quick Post-assessment Survey – Assess Readiness

Results will indicate whether you’re ready to train others, or if there are some topics you need a little more education on. Results will point you to readily available educational modules created by ISTH.

Step 4: Engaging Education – Learn Together

Choose from Interactive Webinars, Podcasts, and Illustrated Clinical Trials.

These educational tools can be accessed by the HTC team members individually or can be used during group education sessions. The tools ensure that all team members have the necessary foundational knowledge prior to engaging in discussions on how to integrate change into practice. Each podcast and webinar is ~30 minutes and is available for AMA PRA Category 1 Credits™.

Clinical Trial Illustrations


BMN 270-301

BMN 270-302

PF 07055480 (SB 525)

PF 06838435 (SPK 9001)

Step 5: Change Guide – Define a Plan

The Change Guide enables you and your care team to explore potential barriers, synergies, and necessary resources needed to make changes in your clinical care approach and to develop actionable plans.


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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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