Pilot and Feasibility Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for MonoMAC
Status:
Completed
Trial end date:
2017-02-26
Target enrollment:
Participant gender:
Summary
Background:
- Stem cells are immature blood cells that grow in the bone marrow and produce all of the
cells needed for normal blood and immunity. Stem cells can be taken from one person
(donor) and given to another person (recipient) through allogeneic stem cell
transplantation. Donor stem cells can then replace the recipients stem cells in the bone
marrow, restoring normal blood production and immunity. Most allogeneic transplants now
use stem cells collected from the donors blood in a process called peripheral blood stem
cell transplantation.
- Monocytopenia and mycobacterial infection (MonoMAC) is an immunodeficiency disease that
is characterized by a lack of monocytes, a type of white blood cell, and an increased
risk of developing mycobacteria infections that may cause tuberculosis.
- Allogeneic stem cell transplantation has been used successfully to treat many kinds of
immune diseases and cancers that develop in blood or immune system cells. Researchers
have been studying a particular kind of stem cell transplantation that uses lower than
usual doses of chemotherapy and particular combinations of drugs to improve the results
of the procedure for patients with blood-related cancers and pre-cancerous conditions.
Objectives:
- To determine the safety and efficacy of reduced-intensity hematopoietic stem cell
transplants (a particular stem cell transplantation procedure) for treating MonoMAC.
Eligibility:
- Patients 18-60 years of age who have MonoMAC and who have been matched with a suitable
stem cell donor.
Design:
- Donors and recipients will undergo separate procedures as part of this protocol.
- Donors:
- National Institutes of Health researchers will take the donor s medical history, perform
a physical exam, take blood samples, and explain the procedure. Tests will be performed
to check the donors heart, lung, kidney, and liver function.
- Donors will receive injections of a drug called filgrastim (G-CSF), which causes stem
cells to travel from bone marrow into blood. The G-CSF shots will be given for 5 to 7
days before the collection procedure.
- Donors will undergo apheresis to collect white blood cells and stem cells directly from
the blood, which can be done as an outpatient procedure. Researchers may consider the
alternative of directly collecting bone marrow from the donor, which will require an
overnight hospital stay.
- Recipients:
- Recipients will receive 3 days of pre-transplant chemotherapy and radiation therapy to
prepare for the transplant. For 4 days before the transplant, recipients will receive
the chemotherapy drug fludarabine, followed by a single dose of radiation therapy, and
will also receive the drugs tacrolimus and sirolimus to prevent the donor cells from
attacking the recipient s normal tissues.
- Recipients will then receive the transplant of donor stem cells and will continue to
receive tacrolimus and sirolimus for 3 months after the transplant to prevent the donor
cells from attacking the recipient s normal tissues. Recipients will be discharged from
the hospital once their condition is stable.
- Recipients will visit the NCI clinic regularly for the first 5 months after the
transplant, and then less often for at least 5 years. Recipients may receive additional
donor immune cells (donor lymphocyte infusion) after the transplant if the study doctors
believe they are needed.