Know Cancer

forgot password

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

2 Years
24 Years
Open (Enrolling)
Hypopituitarism, Hypogonadism, Thyroid Dysfunction, Bone Diseases, Metabolic

Thank you

Trial Information

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

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 screening/training protocol.

As a screening and training study, 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:

(i) 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.

(ii) 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.

(iii) 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.

Inclusion Criteria


- Age greater than or equal to 2 years.

- History of diagnosis of childhood malignancy or HSCT and completion of therapy prior
to entering the study. In order to be eligible for the study, patients must have been
diagnosed with cancer prior to the age of 18. 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. Patients who do not speak
and read English will be excluded from the questionnaire. 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.


-None besides not meeting the inclusion.

Type of Study:


Study Design:


Principal Investigator

Maya B Lodish, M.D.

Investigator Role:

Principal Investigator

Investigator Affiliation:

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)


United States: Federal Government

Study ID:




Start Date:

July 2007

Completion Date:

Related Keywords:

  • Hypopituitarism
  • Hypogonadism
  • Thyroid Dysfunction
  • Bone Diseases, Metabolic
  • Growth Factor
  • Hypothalamic-Pituitary Dysfunction
  • Primary Disorders of the Thyroid
  • Decreased Bone Mineral Density
  • Endocrine Side Effects Related to Cancer Therapy
  • Primary Gonadal Dysfunction
  • Obesity
  • Impaired Glucose Tolerance
  • Thyroid Disorder
  • Cancer Therapy Endocrine Side Effects
  • Pituitary Dysfunction
  • Childhood Cancer Survivors
  • Congenital Abnormalities
  • Bone Diseases
  • Bone Diseases, Metabolic
  • Hypogonadism
  • Hypopituitarism
  • Metabolic Diseases
  • Thyroid Diseases



National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland  20892