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.