Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

Detection and Treatment of Endocrine Abnormalities in Childhood Cancer Survivors and Hematopoietic Stem Cell Transplant Recipients

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 31, 2016 by National Institutes of Health Clinical Center (CC)
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) ) Identifier:
First received: July 18, 2007
Last updated: April 20, 2017
Last verified: October 31, 2016

This study will determine the prevalence of endocrine-related side effects in children who have been treated for cancer and establish a database and registry organized according to cancer diagnosis, treatments and endocrine side effects. In children, the endocrine system, which includes glands and hormones that help to control metabolism, growth, development and reproduction, is particularly vulnerable to long-term side effects associated with cancer and its treatments. The study will also serve to help train medical fellows, residents and students in identifying and managing endocrine abnormalities in children who have been diagnosed with and treated for cancer.

Children between 2 and 24 years of age who have been treated for a childhood cancer and have been disease-free for at least 1 year may be eligible for this study.

All participants undergo the following procedures:

  • Review of cancer treatment record
  • Review of medical and family history
  • Blood draw for DNA studies
  • Physical examination and body measurements (height, weight, waist, body proportions)
  • Completion of child health questionnaires
  • Individualized screening and counseling program
  • Review of the following endocrine systems: growth, pituitary and hypothalamic function, thyroid function, ovary and testicular function, bone health, risk of obesity and diabetes

The following additional studies may be done, as clinically indicated:

  • Magnetic resonance imaging (MRI) of the brain
  • Thyroid, testicular or ovarian ultrasound
  • DEXA scan to measure bone density
  • Wrist x-ray to measure bone age
  • Blood tests
  • Urine pregnancy test for girls who are old enough to have menstrual periods
  • Stimulation testing (tests that involve giving medicine by mouth or in the vein and then measuring blood levels of substances afterwards, such as oral glucose tolerance test, arginine-clonidine growth hormone stimulation test, ACTH stimulation test, and gonadotropin-releasing hormone stimulation test)

Children with endocrine abnormalities are offered standard treatments.

Thyroid Dysfunction
Bone Diseases, Metabolic

Study Type: Observational
Study Design: Time Perspective: Other
Official Title: Detection and Treatment of Endocrine Abnormalities in Childhood Cancer Survivors and Hematopoitic Stem Cell Transplant Recipients

Resource links provided by NLM:

Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 500
Study Start Date: July 13, 2007
Detailed Description:

Endocrine dysfunction is increasingly recognized as one of the most important aspects of quality of life issues, physical and psychosocial development and overall prognosis in pediatric patients diagnosed with neoplasms as well as in patients s/p bone marrow transplant throughout their lifespan. In addition, several of the new, molecularly designed therapies for neoplasms may interact with endocrine signaling; these include receptors and/or their ligands for growth and/or proliferation factors, and disruptors of steroid hormone interactions. The present study serves as a natural history protocol.

As a natural history, this protocol allows our Institute to care for pediatric and adult patients with endocrine related complications associated with prior cancer therapy and/or hematopoietic stem cell transplant (HSCT) for the purposes of:

Training our fellows, residents and students in the identification and management of endocrine abnormalities developing in patients who have been diagnosed with and treated for neoplasms and/or who have received HSCT at the NIH-Clinical center.

Developing new clinical studies for the recognition and therapy of endocrine side effects related to cancer therapy and/or HSCT: this protocol will eventually lead to new, separate protocols that will address specific aspects of endocrinopathies in childhood cancer survivors and HSCT survivors.

The protocol will serve as the basis for outpatient clinics that will function within the context of the pediatric and adult endocrine outpatient clinics: every eligible patient referred to the endocrine service from the NCI, NHLBI, and other NIH institutions and centers will be enrolled in this study, which for the first time will create an endocrine database for these patients. As stated in our aims above, it is our hope that the present investigation will serve as an incubator of further research-focused studies with the ultimate goal of improving the life of children and adults who have been cured of their underlying neoplasms as well as those individuals who are recipients of an HSCT.


Ages Eligible for Study:   2 Years to 24 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Age >2 years.
  • History of diagnosis of malignancy or HSCT and completion of therapy prior to entering the study. Eligible patients must be free of their underlying malignancy for at least one year prior to entering the study, as confirmed by records from the referring oncologist.
  • We will request permission to administer the Child Health Questionnaires (PF 50 and CF87) to all patients who are enrolled in this protocol. This instrument has been validated for use in children ages five and older. Children 10 years and older will complete the child version. The Child Health Questionnaire (CHQ) has been normed in a representative sample in the US, and is being used in large population studies in Australia, Ireland, and the UK. Additionally, it has been rigorously translated into a number of languages using international guidelines including American-Spanish, Canadian-French, Dutch, Finnish, French, German, Italian, Greek, Honduran-Spanish, Mexican-Spanish, Norwegian, Portuguese, and Swedish. For this study will purchase the CHQ in American- Spanish in order to include Spanish-speaking subjects in this part of the study. The costs to purchase the CHQ in numerous languages would be prohibitive for this pilot study. However, if a significant number of subjects in a specific language are recruited, we will consider the purchase of the CHQ for that group of subjects.


  • Metal implants or other ferromagnetic devices, or Foreign material
  • Claustrophobia
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00504218

Contact: Maya B Lodish, M.D. (301) 451-7175

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010   
Sponsors and Collaborators
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Principal Investigator: Maya B Lodish, M.D. Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
  More Information

Additional Information:
Responsible Party: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Identifier: NCT00504218     History of Changes
Other Study ID Numbers: 070192
Study First Received: July 18, 2007
Last Updated: April 20, 2017

Keywords provided by National Institutes of Health Clinical Center (CC):
Growth Factor
Hypothalamic-Pituitary Dysfunction
Primary Disorders of the Thyroid
Decreased Bone Mineral Density
Endocrine Side Effects Related to Cancer Therapy
Primary Gonadal Dysfunction
Impaired Glucose Tolerance
Thyroid Disorder
Cancer Therapy Endocrine Side Effects
Pituitary Dysfunction
Childhood Cancer Survivors

Additional relevant MeSH terms:
Bone Diseases
Bone Diseases, Metabolic
Metabolic Diseases
Gonadal Disorders
Endocrine System Diseases
Musculoskeletal Diseases
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases processed this record on May 25, 2017