Frequently Asked Questions: Pediatric AIDS Research

What is the National Institutes of Health (NIH)?

NIH is one of eight health agencies of the Public Health Service, which is part of the U.S. government's Department of Health and Human Service (DHHS). NIH is comprised of 24 institutes, centers, and divisions. It has 75 buildings on more than 300 acres on its main campus in Bethesda, Maryland, just outside of Washington, D.C. The NIH mandate is to acquire new knowledge that will help prevent, detect, diagnose, and treat human disease and disability.

What kinds of treatment are offered for HIV-infected children?

In the Pediatric HIV Clinical Research Program, we are evaluating new treatments for HIV infection, AIDS, and related illnesses in children. These evaluations of new treatments are referred to as clinical trials or clinical treatment protocols. Before new treatments can be used in children they must undergo extensive laboratory research and sufficient testing in animals and adults to deem them safe to try in children. Each treatment protocol undergoes a rigorous review by many different people and groups, including the Food and Drug Administration (FDA), the NCI Institutional Review board (IRB) and others.

The clinical trials or protocols in the PHWG program are generally Phase I or Phase I/II: Phase I studies attempt to determine the safest or best (optimal) dose of the experimental drug or combination of drugs, the behavior of the drug(s) in the human body, and its side effects or toxicities.

Phase II studies evaluate the ability of the drug(s) to produce measurable positive effects in HIV disease, i.e., an increase in CD4 count or a decrease in viral load.

Who is eligible to receive these treatments?

HIV-infected children and adolescents ages 2 months to 21 years, who are U.S. citizens, and who meet protocol-specific criteria for enrollment.

How is a referral initiated?

Referral can be initiated by the child's parent or health care provider by contacting the PHWG Referral Coordinator at (301) 402-1391. A Referral Information Form is sent to the child's primary/referring physician, an initial telephone screen is done, and, if the child is potentially eligible for one or more of the currently available protocols, an appointment is scheduled for an evaluation. While parents can initiate the referral, it is advised that they discuss their interest in participating in a research protocol with the child's physician and request him/her to complete the Referral Information Form. It is required that the child have a referral from a physician who will continue to provide acute and routine medical care during the time the child participates in NIH studies.

What is required for the initial evaluation?

• a letter from the primary/referring physician indicating that he/she wishes to refer the patient
  
  
• a completed Referral Information Form and pertinent medical information
  
  
• recent laboratory results including a listing of all known CD4 counts and viral load levels

What is included in the initial evaluation?

• comprehensive medical history and physical exam
• laboratory evaluation, including CD4 count and viral load level
• neurologic evaluation
• chest x-ray, electrocardiogram (EKG), and echocardiogram
• eye examination
• dental evaluation
• physical or occupational evaluation
• neurocognitive testing
• imaging studies-brain MRI and CT scans

Additional evaluations may also be required for certain protocols.

How is the referring/primary physician's role in the patient's care maintained and supported while a patient is participating in a clinical protocol at NIH?

The primary/referring physician is a critical member of the PHWG research team. In addition to providing the child's physician with detailed medical information, the research team also consults with the physician when there are particular concerns or issues related to the child's study participation, drug administration, or monitoring of clinical toxicities.

How is protocol eligibility determined?

Each protocol has very strict rules (eligibility criteria) which we must follow in order to enroll a child in a study. These eligibility criteria are determined very early in the development of a protocol, as each protocol is designed to answer specific scientific questions. The eligibility criteria consist of both inclusion criteria (conditions which must be present for the child to enroll) and exclusion criteria (conditions which must not be present if the child is to enroll). After a child has completed the initial evaluation, we examine the results of his/her evaluation as well as the medical history to determine whether the child meets the inclusion and exclusion criteria.

Can the parent or referring/primary physician choose the protocol they prefer if the child is eligible for more than one study?

Often parents have heard about new treatments they would like their child to receive. If the child is eligible for more than one protocol, protocol assignment is made by the attending physician , the clinical team, and the family.

What happens if it is determined that a child is not eligible for a study after completing the screening evaluation?

If a child is not eligible for a study, we will offer the best advice we can regarding currently available treatment, and we will also make a treatment recommendation to the referring/primary physician. A child can be referred again at a later date if he experiences a change in clinical status which is likely to make him eligible.

What does treatment at NIH cost?

There is no charge to patients for the evaluations, treatments, or medical services provided at NIH as part of their participation in clinical research studies. In addition, to facilitate participation in protocols, airfare is provided to and from NIH and lodging is provided at The Children's Inn.

FOR ADDITIONAL INFORMATION AND PATIENT REFERRAL, PLEASE CALL (301) 402-1391 OR E-MAIL: PedHAMBref@mail.nih.gov

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