The research goal of this study is to obtain CD34+ hematopoietic stem cells (HSC) from peripheral blood and/or bone marrow, and Mononuclear Cells (lymphocytes and monocytes), and granulocytes (grans) from peripheral blood that will be used in the laboratory and/or in the clinic to develop new cell therapies for patients with inherited or acquired disorders of immunity or blood cells. Development of novel cellular therapies requires access to HSC, Mononuclear Cells and/or granulocytes as the essential starting materials for the pre-clinical laboratory development of gene therapies and other engineered cell products. HSC or blood cells from healthy adult volunteers serve both as necessary experimental controls and also as surrogates for patient cells for clinical scale-up development. HSC or blood cells from patients serve both as the necessary experimental substrate for novel gene therapy and cellular engineering development for specific disorders and as pre-clinical scale up of cellular therapies. Collection of cells from adult patients collected in the NIH Department of Transfusion Medicine (DTM) under conditions conforming to accepted blood banking clinical practice may also be used directly in or cryopreserved for future use in other NIH protocols that have all required regulatory approvals allowing such use. In summary, the research goal of this protocol is the collection of HSC or blood cells that may be used for both laboratory research and/or for clinical treatment in other approved protocols.
Patients will have a genetically defined PID or have a clinical history of recurrent infections or other problems suggestive of PID or other blood disorder, must be 18-70 years of age,
Some patients may have active bacterial or fungal infection at the time of study entry.
Preserved renal function (creatinine less than or equal to 2.5 mg/dL; less than or equal to 3+ proteinuria); preserved hepatic function (bilirubin less than or equal to 2.0 mg/dl); preserved hematologic function (WBC greater than or equal to1000/mm^3;granulocytes greater than or equal to 500/mm^3; platelets greater than or equal to 100,000; hematocrit greater than or equal to 25). Of note, patients with PID often have associated chronic thrombocytopenia. Patients with stable chronic thrombocytopenia will be eligible for collection, at the investigator s discretion, with the caveat that patients with platelet count <40,000 the day prior to collection will be transfused with platelets on the morning of collection. Platelets may also be given to these patients following the collection if medically indicated..
Patients of childbearing potential may be entered if using effective contraception and having a negative serum or urine pregnancy test within one week of beginning G-CSF administration.
None
Patients with PID or other blood disorders Healthy adult individuals volunteering for research donation
Cohort
Other
Not listed
Mobilize to peripheral blood and apheresis collect CD34+PBSC of PID patients for clinical treatment and in part (or entirely) for laboratory research
1\. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC patients with any inherited primary immune deficiency (PID) or other blood disorders where PBSC from these patients may be designated entirely or in part for future clinical treatment of the patient
Mobilize to the peripheral blood and apheresis collect CD34+PBSC from healthy volunteers for research
2\. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC from healthy volunteers, which will be designated entirely for laboratory research.
Mobilize to peripheral blood mononuclear cells and granulocytes
3.To efficiently and safely collect peripheral blood mononuclear cells and granulocytes from adult subjects with chronic granulomatous disease or other PID affecting granulocyte function, or from adult healthy donors volunteers for preclinical cell therapy studies, following either no stimulation, G-CSF or dexamethasone alone, or combined single dose G-CSF and dexamethasone as per DTM protocol depending on the number of cells desired.
bone marrow harvest
4.To perform a bone marrow harvest by needle aspiration using standard of medical care methods from adult patients with PID or other blood disorder in sufficient quantity cryopreserved in CC DTM to serve as the source of autologous HSC for gene transfer ex vivo transduction if that patient is enrolled in a separate IRB approved protocol of gene transfer treatment designed to treat the genetically defined type of PID affecting that patient; and the patient has been unable to mobilize and have collected sufficient PBSC to qualify for treatment in that protocol.
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