Patient Support & Resources

Recently, Children’s Hospital Boston received grant funding from the National Institutes of Health (NIH) to establish the first nationwide patient registry and tissue repository for Pediatric MDS and bone marrow failure disorders. This will allow researchers at Children’s and collaborating institutions to collect information and tissue samples to help us better understand this condition.

Ultimately, we hope to identify new therapies for this condition which is currently only cured with a bone marrow transplant.

In the following pages, you’ll find more information about our registry, instructions on how to join the registry, and recommended resources.

To learn more about participating in the registry, please call Dr. Inga Hofmann at 888-5-PediMDS.

The Pediatric Myelodysplastic Syndrome (MDS) and Bone Marrow Failure (BMF) Registry is a national research initiative focused on improving the understanding, diagnosis and treatment of MDS and BMF disorders. We do not know for certain whether this registry will have any direct benefit to you or your child. However, by joining the registry, you are contributing to research that will help future patients.

The general process to joining the registry is outlined below. We are available at any time to answer questions about the registration process and program goals. Patients interested in the registry can obtain additional information about the research study, including a copy of the informed consent, by contacting Dr. Inga Hofmann at 888-5-PediMDS.

What is involved in participating in the Pediatric MDS and BMF Registry?

After signed informed consent is obtained, we will collect information from medical records, laboratory reports and pathology reports with the help of your primary care provider (typically your Hematologist/Oncologist). Clinical and research samples are obtained when blood, bone marrow or tissue biopsies are obtained for your routine clinical care to avoid extra needle sticks or procedures.

Physician Referral

To join the registry, your children's referring physician should contact principal investigator Dr. Inga Hofmann by calling us at 888-5-PediMDS to register the patient, which includes the transfer of clinical and diagnostic data.

Patients and families will receive a consent form and have the ability to ask any questions they have before joining. Referring physicians can request this referral packet, which includes instructions on information we need to complete a registration.

To download a letter to your referral physician, click here.

For specific instructions on how to join the registry, click here.

Find a Study Center near you, click here.

For more useful information and resources on MDS and BMF, we recommend the following websites:

  • Myelodysplastic Syndrome Specialty Care, Children’s Hospital Boston
  • Fanconi Anemia & Bone Marrow Failure Multidisciplinary Clinic, Children’s Hospital Boston
  • European Working Group of MDS and JMML in Childhood (EWOG-MDS)MDS
  • Foundation
  • Aplastic Anemia & MDS International Foundation
  • MDS Information, National Institutes of Health