Shwachman Diamond Syndrome Registry and Study

Description

Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition. SDS-Like patients share clinical features with SDS but lack mutations in known SDS genes. Since SDS/SDS-Like syndromes are rare diseases, data are sparse regarding the clinical features, natural history, clinical outcomes with current management, and treatment. For this reason, the SDS Registry was formed to collect clinical data from medical records and to bank biological samples with the goal of understanding SDS/SDS-Like diseases to develop better treatments and improve the health of patients with these conditions.

Conditions

Shwachman-Diamond Syndrome, Shwachman-Diamond Syndrome-Like

Study Overview

Study Details

Study overview

Shwachman-Diamond syndrome (SDS) is a genetic condition characterized by bone marrow failure, medical co-morbidities, and leukemia predisposition. SDS-Like patients share clinical features with SDS but lack mutations in known SDS genes. Since SDS/SDS-Like syndromes are rare diseases, data are sparse regarding the clinical features, natural history, clinical outcomes with current management, and treatment. For this reason, the SDS Registry was formed to collect clinical data from medical records and to bank biological samples with the goal of understanding SDS/SDS-Like diseases to develop better treatments and improve the health of patients with these conditions.

Shwachman Diamond Syndrome Registry and Study (SDS Registry)

Shwachman Diamond Syndrome Registry and Study

Condition
Shwachman-Diamond Syndrome
Intervention / Treatment

-

Contacts and Locations

Aurora

Children's Hospital Colorado, Aurora, Colorado, United States, 80045

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Boston

Dana-Farber Cancer Institute, Boston, Massachusetts, United States, 02115

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Biallelic mutations in SBDS, or pathogenic mutations in DNAJC21, EFL1, or SRP54 OR
  • * Shwachman-Diamond Syndrome defined clinically OR
  • * Clinically suspected Shwachman-Diamond Syndrome OR
  • * Phenotypic features suggestive of SDS OR
  • * Parents, siblings, and other blood relatives of any age, living and deceased, of patients with SDS or SDS-Like conditions are eligible for this study

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Boston Children's Hospital,

Study Record Dates

2090-01-01