This study is designed to allow the evaluation, follow-up, and medical care of patients with
blood disorders not currently participating in a research study being conducted by the
Hematology Branch of the National Heart, Lung and Blood Institute (NHLBI) or not being
screened for participation in a study.
The purpose of this study is to allow investigation into the blood disorders of patients in
order to teach, learn, and gather more information about diseases of the blood.
In addition, this study allows researchers the opportunity to evaluate patients referred to
the Hematology Branch of the NHLBI with rare or undiagnosed diseases of the blood. This may
be potentially beneficial to the patient and at the same time contribute to the development
of new research ideas.
This protocol is designed to allow the evaluation, follow up, and standard medical care of
patients (and when appropriate, their stem cell donor) with bone marrow failure states,
cytopenias, metastatic solid tumors, or hematologic malignancies not currently entered on an
active NHLBI Hematology Branch protocol or not being screened for an active NHLBI research
protocol. Its purpose is to allow investigation into the hematologic problems of these
patients for the primary purpose of teaching and furthering general hematologic knowledge.
The ability to evaluate and treat patients with a wide variety of hematologic disease is
critical to maintaining our accreditation as a hematology fellowship program and training our
fellows to be competent hematologists, as well as for keeping all staff, including senior
staff, up-to-date and familiar with the evaluation and treatment of patients with a wide
spectrum of hematologic disease. It also allows the Hematology Branch to evaluate patients
referred with rare or as yet undiagnosed hematologic problems, both potentially benefiting
the patient and stimulating new research directions for the Hematology Branch in the future.
Periodic follow-up and treatment of patients previously entered on Hematology Branch
protocols in order to monitor the long-term course of the underlying hematologic state and
the consequences of experimental treatments is also required for fellowship training, and for
maintenance of good referral relationships with patients and their physicians.
- INCLUSION CRITERIA:
Patients (and when appropriate the patient's stem cell donor) may be entered on this
protocol at the discretion of the Principal and/or Associate Investigators if :
An investigator decides that it is in the best interest of the patient/donor and the Branch
for the patient to be enrolled in this protocol while awaiting a decision on eligibility
for other protocols; or after enrollment on another protocol, or to allow careful
evaluation of patients with unique or rare disorders of direct interest to the Branch; or
with more common disorders for fellowship teaching purposes.
The patient or the patient's guardian is capable of informed consent and signs the consent
form. The consent form will be signed by parents or guardians of patients under the age of
Age greater than or equal to 2 and weight greater than 12 kg.
Georg Aue, M.D.
National Heart, Lung, and Blood Institute (NHLBI)
A. John Barrett, M.D.
Phone: (301) 402-4170