PID, Primary Immunodeficiency
Conditions
Keywords
end-stage lung disease, Bilateral Orthotopic Lung Transplant (BOLT) candidate, CD3/CD19 negative allogeneic hematopoietic stem cells (HSCT), CD3+/CD19+ depleted Bone Marrow Transplant (BMT), Cadaveric (Deceased) Donor, Unrelated (Deceased) Donor
Brief summary
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched CD3+/CD19+ depleted bone marrow transplant (BMT) is safe and effective for individuals aged 10 through 45 years with the diagnosis of primary immunodeficiency (PID) and end-stage lung disease. The enrollment goal: 8 participants who receive both BOLT and BMT.
Detailed description
The primary purpose of this study is to evaluate the safety and efficacy of performing bilateral orthotopic lung transplantation (BOLT) followed by cadaveric, partially HLA-matched CD3+/CD19+-depleted hematopoietic stem cell transplantation (HSCT) from the same donor for participants with primary immunodeficiency diseases (PID) and end-stage lung disease. For many patients with primary immunodeficiencies, HSCT, which we refer to as Bone Marrow Transplant (BMT) is a curative, life-saving therapy, resulting in restoration of function in the immune system. Patients with primary immunodeficiencies often develop pulmonary complications as a result of chronic or recurrent infections, making them ineligible for BMT due to the high risk of mortality and pulmonary complications. Lung transplant prior to BMT would allow for restoration of pulmonary function prior to BMT, allowing PID patients to proceed to BMT , which would be curative for the patient's underlying immunodeficiency. As a secondary aim, after successful engraftment with donor bone marrow, the feasibility of participants tolerating planned withdrawal of immunosuppression and achieving eventual freedom from all immunosuppressive drugs and attaining a tolerant state will be assessed. This is a single center study in which participants receive a cadaveric, partially Human Leukocyte Antigen (HLA)-matched lung transplant followed by a CD3+/CD19+ depleted bone marrow transplant (BMT) from the same donor. In this study, the investigators will use a ≥ 2/6 HLA-matched T cell depleted bone marrow transplant from a cadaveric organ donor with an identical ABO blood type as the recipient. Participants will undergo: * Bilateral orthotopic lung transplant (BOLT) utilizing basiliximab induction or an alternate induction therapy based on their underlying disease. Rituximab may be initiated prior to the lung transplant, with tacrolimus as the ongoing maintenance immunosuppression. * BMT utilizing CD3+/CD19+-depleted bone marrow with bone marrow conditioning beginning no less than 8 weeks after BOLT. The duration of participant involvement in the trial is up to 2 years post-BMT.
Interventions
BIOLOGICALCD3/CD19 neg allogeneic BMT
Negative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection, and cryopreserved for later marrow transplantation. BMT conditioning and transplantation will start at least 8 weeks or more post lung transplant - once the participant is clinically judged suitable to undergo BMT conditioning and transplantion.
Sponsors
University of Pittsburgh
PPD Development, LP
National Institute of Allergy and Infectious Diseases (NIAID)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to 45 Years
Healthy volunteers
No
Inclusion criteria
* Subject and/or parent guardian must be able to understand and provide informed consent; * Subject fulfills criteria for United Network of Organ Sharing (UNOS) listing; * Subject must have evidence of an underlying primary immunodeficiency for which Bone Marrow Transplant (BMT) is clinically indicated. Examples of such diseases include, but are not limited to: * Severe Combined Immunodeficiency (SCID) * Combined immunodeficiency with defects in T-cell-mediated immunity, including Omenn syndrome and DiGeorge Syndrome * Severe Chronic Neutropenia * Chronic Granulomatous Disease (CGD) * Hyper Immunoglobulin E (IgE) Syndrome or Job's Syndrome * CD40 or CD40L deficiency * Wiskott-Aldrich Syndrome * Mendelian Susceptibility to Mycobacterial Disease * GATA2-associated Immunodeficiency. * Subjects must have evidence of end-stage lung disease and be candidates for bilateral orthotopic lung transplant as determined by the lung transplant team; * Glomerular filtration rate (GFR) ≥ 50 mL/min/1.73 m\^2; * Aspartate aminotransferase (AST), Alanine aminotransaminase (ALT) ≤ 4x upper limit of normal, total bilirubin ≤ 2.5 mg/dL, normal INR; * Cardiac ejection fraction ≥ 40% or shortening fraction ≥ 26%; * Negative pregnancy test for females \>10 years old or who have reached menarche, unless surgically sterilized; * All females of childbearing potential and sexually active males must agree to use a Food and Drug Administration (FDA) approved method of birth control for up to 24 months after BMT or for as long as they are taking any medication that may harm a pregnancy, an unborn child or may cause birth defect; and * Subject and/or parent guardian will also be counseled regarding the potential risks of infertility following BMT and advised to discuss sperm banking or oocyte * harvesting. Eligibility for Bone Marrow Transplant\*: * GFR \>50 mL/min/1.73 m\^2; * AST, ALT \<4x upper limit of normal, Total bilirubin \< 2.5 mg/dL; * Cardiac ejection fraction ≥40% or shortening fraction of at least 26%; * Human Immunodeficiency Virus (HIV) negative by serology and PCR; * Human T-lymphotropic virus (HTLV) serology negative; * Forced vital capacity (FVC) and Forced expiratory volume (FEV1) ≥ 40% predicted for age and SpO2 of \>90% at rest on room air AND with clearance by the lung transplant team; * Absence of uncontrolled infection as determined by blood cultures and radiographic results of previously affected sites, in particular, pulmonary densities during the past 2 weeks prior to chemotherapy; * Absence of Acute Cellular Rejection (ACR); and * Bone marrow processing has been completed, and an appropriate stem cell product is available for administration. * Note: The decision to proceed with the BMT will be at the discretion of the lung transplant team following clearance by the bone marrow team based on the criteria below. The conditioning for the BMT will begin no less than 8 weeks following the lung transplant.
Exclusion criteria
* Inability or unwillingness of a participant to give written informed consent or comply with study protocol; * Subjects who have underlying malignant conditions; * Subjects who have non-malignant conditions that do not require hematopoietic stem cell transplantation; * Human Immunodeficiency Virus (HIV) positive by serology or polymerase chain reaction (PCR), human T-lymphotropic virus (HTLV) positive by serology; * Females who are pregnant or who are lactating; * Allergy to dimethyl sulfoxide (DMSO) or any other ingredient used in the manufacturing of the stem cell product; * Uncontrolled pulmonary infection, as determined by radiographic findings and/or significant clinical deterioration. \-- Pulmonary colonization with multiple organisms is common, and will not be considered an exclusion criterion. * Uncontrolled systemic infection, as determined by the appropriate confirmatory testing e.g. blood cultures, PCR testing, etc.; * Recent recipient of any licensed or investigational live attenuated vaccine(s), within 4 weeks of transplant; or * Past or current medical problems or findings from physical examination or laboratory testing that are not listed above, which, in the opinion of the investigator, may pose: * additional risks from participation in the study, * may interfere with the participant's ability to comply with study requirements, * or that may impact the quality or interpretation of the data obtained from the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety: Death | Average of approximately 31 months for those who received an initial transplant | How many, if any, participants die during study participation. |
| Safety: Engraftment Syndrome | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | How many, if any, participants develop engraftment syndrome. |
| Engraftment Failure | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | — |
| Grade 4 or 5 Events Potentially Attributable to Rituximab | Average of ~25 months. | — |
| BOS at 1 Year Post BOLT | throughout the first year post BOLT | Diagnosis of BOS (Bronchiolitis Obliterans Syndrome) at any time up to 1 year post BOLT. BOS is one of the undesirable complications of lung transplantation. |
| Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support | at 1 Year Post Lung Transplant (BOLT) | The number of participant(s) who need either supplemental oxygen and/or ventilator support (noninvasive/invasive) will be assessed using the Bronchiolitis Obliterans Syndrome (BOS) Classification Score for pulmonary function (e.g., the Forced Expiratory Volume in 1 Second (FEV1). FEV1 is air volume exhaled in 1 second during spirometry, a lung function test. (Continuing dependence on supplemental oxygen or ventilatory support is an undesirable outcome of lung transplantation). |
| Efficacy: Count of Participants With T-cell Chimerism | 1 Year Post Bone Marrow Transplant (BMT) | The number of participants who have ≥ 25% donor T-cell chimerism. |
| Efficacy: Count of Participants With Myeloid Chimerism | 1 Year Post Bone Marrow Transplant (BMT) | The number of participants with myeloid disorders (e.g. Chronic Granulomatous Disease \[CGD\]) who attain ≥ 10% myeloid chimerism. |
| Efficacy: Count of Participants B-cell Chimerism | 1 Year Post Bone Marrow Transplant (BMT) | The number of participants with B-cell disorders who attain ≥ 10% B-cell chimerism. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Count of Participants With Chronic Graft-Versus-Host Disease (GVHD) | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | The number of participants who develop chronic graft-versus-host disease (GVHD) following tandem BOLT and BMT. |
| Count of Participants Able to Proceed to BMT | 6 Months Post Lung Transplant (BOLT) | The number of participants for which it is feasible to proceed to BMT within 6 months following lung transplant. |
| Count of Pre-BMT Conditioning Rituximab Related Adverse Events | Average of approximately 9 months. | The number of Grade 4 or 5 adverse events possibly related to the use of rituximab prior to the start of BMT conditioning. |
| Count of Participants Who Develop Chronic Lung Allograft Dysfunction or Allograft Failure | up to 2 years post BOLT | A significant development in chronic lung allograft dysfunction (as evidenced by a change in BOS stage) or allograft failure at 1 year and up to 2 year post lung transplant (for lung transplant alone and BOLT-BMT subjects. |
| Count of Participants Who Achieve Tolerance | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | The number of participants who develop tolerance to both the host and pulmonary graft. Definition of tolerance: A participant's successful withdrawal from systemic immunosuppression for 6 weeks with no increase cGVHD score and stable or improving PFTs. |
| Long Term Complications of Combined Solid Organ and Bone Marrow Transplant | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | Summary of long-term complications of combined solid organ and bone marrow transplant (BMT). |
| Count of Participants Who Develop Acute Cellular Rejection and Graft Failure | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | The number of participants who develop acute cellular rejection and graft failure post BMT. |
| Count of Participants Able to Initiate Withdrawal of Immunosuppression | 1 year following BMT | The number of participants who are able to start immunosuppression withdrawal. |
| Time to Withdrawal of Immunosuppression | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | Time from BMT to withdrawal of immunosuppression. |
| Time to Independence From Treatment Dose Antimicrobial Drug | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | A measure of pathogen-specific immunity. |
| Post-BMT Achievement of Normal Lymphocyte Count for T-cell Lymphopenias | 1 year post BMT | For participants with immune deficiencies with T cell lymphopenias, number of participants achieving age adjusted, low limit normal range lymphocyte count by 1-year post-BMT. |
| Count of Participants Who Develop Acute Graft-Versus-Host Disease (GVHD) | Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants. | The number of participants who develop acute graft-versus-host disease (GVHD) following tandem BOLT and BMT. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| BOLT-BMT Protocol Participants may receive a double lung transplant followed by a bone marrow (hematopoietic stem cells) transplant, if a partially HLA-matched organ offer is accepted. The lungs and allogeneic hematopoietic stem cells will be from the same partially HLA-matched cadaveric donor. Prior to marrow transplantation, the marrow will be negatively selected for CD3/CD19 using a CliniMACS® depletion device.
CD3/CD19 neg allogeneic BMT: Negative selection for CD3/CD19 will be performed on a CliniMACS® depletion device within 36 hours of collection, and cryopreserved for later marrow transplantation. BMT conditioning and transplantation will start at least 8 weeks or more post lung transplant - once the participant is clinically judged suitable to undergo BMT conditioning and transplantion. | 5 |
| Total | 5 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 3 |
| Overall Study | No longer eligible to receive transplants or study interventions due to medical contraindication | 1 |
Baseline characteristics
| Characteristic | BOLT-BMT Protocol |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 5 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Primary Immunodeficiency B-cell disorder (e.g. CVID) | 2 Participants |
| Primary Immunodeficiency Myeloid disorder (e.g. CGD) | 1 Participants |
| Primary Immunodeficiency T-cell lymphopenia/T-cell disorder (SCID, CID) | 2 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 4 Participants |
| Region of Enrollment United States | 5 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 4 / 5 |
| other Total, other adverse events | 5 / 5 |
| serious Total, serious adverse events | 5 / 5 |
Outcome results
Primary
BOS at 1 Year Post BOLT
Diagnosis of BOS (Bronchiolitis Obliterans Syndrome) at any time up to 1 year post BOLT. BOS is one of the undesirable complications of lung transplantation.
Time frame: throughout the first year post BOLT
Population: Recipients of CD3/CD19-depleted marrow (includes 1 participant receiving BMT \>1 year post BOLT)
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | BOS at 1 Year Post BOLT | 0 Participants |
Primary
Efficacy: Count of Participants B-cell Chimerism
The number of participants with B-cell disorders who attain ≥ 10% B-cell chimerism.
Time frame: 1 Year Post Bone Marrow Transplant (BMT)
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Efficacy: Count of Participants B-cell Chimerism | 0 Participants |
Primary
Efficacy: Count of Participants With Myeloid Chimerism
The number of participants with myeloid disorders (e.g. Chronic Granulomatous Disease \[CGD\]) who attain ≥ 10% myeloid chimerism.
Time frame: 1 Year Post Bone Marrow Transplant (BMT)
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Efficacy: Count of Participants With Myeloid Chimerism | 1 Participants |
Primary
Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support
The number of participant(s) who need either supplemental oxygen and/or ventilator support (noninvasive/invasive) will be assessed using the Bronchiolitis Obliterans Syndrome (BOS) Classification Score for pulmonary function (e.g., the Forced Expiratory Volume in 1 Second (FEV1). FEV1 is air volume exhaled in 1 second during spirometry, a lung function test. (Continuing dependence on supplemental oxygen or ventilatory support is an undesirable outcome of lung transplantation).
Time frame: at 1 Year Post Lung Transplant (BOLT)
Population: Recipients of CD3/CD19-depleted marrow (includes 1 participant receiving BMT \>1 year post BOLT)
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support | 0 Participants |
Primary
Efficacy: Count of Participants With T-cell Chimerism
The number of participants who have ≥ 25% donor T-cell chimerism.
Time frame: 1 Year Post Bone Marrow Transplant (BMT)
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Efficacy: Count of Participants With T-cell Chimerism | 1 Participants |
Primary
Engraftment Failure
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Engraftment Failure | 1 Participants |
Primary
Grade 4 or 5 Events Potentially Attributable to Rituximab
Time frame: Average of ~25 months.
Population: All participants
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Grade 4 or 5 Events Potentially Attributable to Rituximab | 4 Participants |
Primary
Safety: Death
How many, if any, participants die during study participation.
Time frame: Average of approximately 31 months for those who received an initial transplant
Population: This Number of Participants analyzed includes one participant who became ineligible: no longer eligible to receive transplants or study interventions due to medical contraindication. One participant was terminated from the study prior to receiving a study transplant, prior to developing fatal illness, because this patient developed an infection which made her ineligible to proceed to transplant. This participant died during follow-up of safety events on study after termination from the study.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Safety: Death | 4 Participants |
Primary
Safety: Engraftment Syndrome
How many, if any, participants develop engraftment syndrome.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Safety: Engraftment Syndrome | 0 Participants |
Secondary
Count of Participants Able to Initiate Withdrawal of Immunosuppression
The number of participants who are able to start immunosuppression withdrawal.
Time frame: 1 year following BMT
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants Able to Initiate Withdrawal of Immunosuppression | 0 Participants |
Secondary
Count of Participants Able to Proceed to BMT
The number of participants for which it is feasible to proceed to BMT within 6 months following lung transplant.
Time frame: 6 Months Post Lung Transplant (BOLT)
Population: Recipients of a bilateral lung transplant
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants Able to Proceed to BMT | 1 Participants |
Secondary
Count of Participants Who Achieve Tolerance
The number of participants who develop tolerance to both the host and pulmonary graft. Definition of tolerance: A participant's successful withdrawal from systemic immunosuppression for 6 weeks with no increase cGVHD score and stable or improving PFTs.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants Who Achieve Tolerance | 0 Participants |
Secondary
Count of Participants Who Develop Acute Cellular Rejection and Graft Failure
The number of participants who develop acute cellular rejection and graft failure post BMT.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants Who Develop Acute Cellular Rejection and Graft Failure | 0 Participants |
Secondary
Count of Participants Who Develop Acute Graft-Versus-Host Disease (GVHD)
The number of participants who develop acute graft-versus-host disease (GVHD) following tandem BOLT and BMT.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants Who Develop Acute Graft-Versus-Host Disease (GVHD) | 1 Participants |
Secondary
Count of Participants Who Develop Chronic Lung Allograft Dysfunction or Allograft Failure
A significant development in chronic lung allograft dysfunction (as evidenced by a change in BOS stage) or allograft failure at 1 year and up to 2 year post lung transplant (for lung transplant alone and BOLT-BMT subjects.
Time frame: up to 2 years post BOLT
Population: Recipients of a bilateral lung transplant
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants Who Develop Chronic Lung Allograft Dysfunction or Allograft Failure | 0 Participants |
Secondary
Count of Participants With Chronic Graft-Versus-Host Disease (GVHD)
The number of participants who develop chronic graft-versus-host disease (GVHD) following tandem BOLT and BMT.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Participants With Chronic Graft-Versus-Host Disease (GVHD) | 0 Participants |
Secondary
Count of Pre-BMT Conditioning Rituximab Related Adverse Events
The number of Grade 4 or 5 adverse events possibly related to the use of rituximab prior to the start of BMT conditioning.
Time frame: Average of approximately 9 months.
Population: All participants, prior to the start of BMT Conditioning
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Count of Pre-BMT Conditioning Rituximab Related Adverse Events | 3 Participants |
Secondary
Long Term Complications of Combined Solid Organ and Bone Marrow Transplant
Summary of long-term complications of combined solid organ and bone marrow transplant (BMT).
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Long Term Complications of Combined Solid Organ and Bone Marrow Transplant | 0 Participants |
Secondary
Post-BMT Achievement of Normal Lymphocyte Count for T-cell Lymphopenias
For participants with immune deficiencies with T cell lymphopenias, number of participants achieving age adjusted, low limit normal range lymphocyte count by 1-year post-BMT.
Time frame: 1 year post BMT
Population: Recipients of CD3/CD19-depleted marrow
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BOLT-BMT Protocol | Post-BMT Achievement of Normal Lymphocyte Count for T-cell Lymphopenias | 0 Participants |
Secondary
Time to Independence From Treatment Dose Antimicrobial Drug
A measure of pathogen-specific immunity.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: None of the participants achieved independence from treatment dose antimicrobial drugs during study participation.
Secondary
Time to Withdrawal of Immunosuppression
Time from BMT to withdrawal of immunosuppression.
Time frame: Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Population: None of the participants withdrew from IS during study participation.