Acute Myeloid Leukemia Clinical Trial
Official title:
A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic Syndrome (MDS)
Verified date | April 2024 |
Source | Children's Oncology Group |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This phase III trial compares hematopoietic (stem) cell transplantation (HCT) using mismatched related donors (haploidentical [haplo]) versus matched unrelated donors (MUD) in treating children, adolescents, and young adults with acute leukemia or myelodysplastic syndrome (MDS). HCT is considered standard of care treatment for patients with high-risk acute leukemia and MDS. In HCT, patients are given very high doses of chemotherapy and/or radiation therapy, which is intended to kill cancer cells that may be resistant to more standard doses of chemotherapy; unfortunately, this also destroys the normal cells in the bone marrow, including stem cells. After the treatment, patients must have a healthy supply of stem cells reintroduced or transplanted. The transplanted cells then reestablish the blood cell production process in the bone marrow. The healthy stem cells may come from the blood or bone marrow of a related or unrelated donor. If patients do not have a matched related donor, doctors do not know what the next best donor choice is. This trial may help researchers understand whether a haplo related donor or a MUD HCT for children with acute leukemia or MDS is better or if there is no difference at all.
Status | Recruiting |
Enrollment | 435 |
Est. completion date | December 1, 2027 |
Est. primary completion date | December 1, 2027 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 6 Months to 21 Years |
Eligibility | Inclusion Criteria: - PATIENT INCLUSION CRITERIA FOR ENROLLMENT: - 6 months to < 22 years at enrollment - Diagnosed with ALL, AML, or MDS or mixed phenotype acute leukemia (MPAL) for which an allogeneic hematopoietic stem cell transplant is indicated. Complete Remission (CR) status will not be confirmed at the time of enrollment. CR as defined in these sections is required to proceed with the actual HCT treatment plan - Has not received a prior allogeneic hematopoietic stem cell transplant - Does not have a suitable human leukocyte antigen (HLA)-matched sibling donor available for stem cell donation - Has an eligible haploidentical related family donor based on at least intermediate resolution HLA typing - Patients who also have an eligible 8/8 MUD adult donor based on confirmatory high resolution HLA typing are eligible for randomization to Arm A or Arm B. - Patients who do not have an eligible MUD donor are eligible for enrollment to Arm C - All patients and/or their parents or legal guardians must sign a written informed consent - All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met - Co-Enrollment on other trials - Patients will not be excluded from enrollment on this study if already enrolled on other protocols for treatment of high risk and/or relapsed ALL, AML, MPAL and MDS. This is including, but not limited to, COG AAML1831, COG AALL1821, the EndRAD Trial, as well as local institutional trials. We will collect information on all co-enrollments - Patients will not be excluded from enrollment on this study if receiving immunotherapy prior to transplant as a way to achieve remission and bridge to transplant. This includes chimeric antigen receptor (CAR) T cell therapy and other immunotherapies - PATIENT INCLUSION CRITERIA TO PROCEED TO HCT: - Karnofsky Index or Lansky Play-Performance Scale >= 60 on pre-transplant evaluation. Karnofsky scores must be used for patients >= 16 years of age and Lansky scores for patients =< 16 years of age (within 4 weeks of starting therapy) - A serum creatinine based on age/gender as follows: 6 months to < 1 year: 0.5 mg/dL (Male); 0.5 mg/dL (Female) 1. to < 2 years: 0.6 mg/dL (Male); 0.6 mg/dL (Female) 2. to < 6 years: 0.8 mg/dL (Male); 0.8 mg/dL (Female) 6 to < 10 years: 1 mg/dL (Male); 1 mg/dL (Female) 10 to < 13 years: 1.2 mg/dL (Male); 1.2 mg/dL (Female) 13 to < 16 years: 1.5 mg/dL (Male); 1.4 mg/dL (Female) >= 16 years: 1.7 mg/dL (Male); 1.4 mg/dL (Female) - OR - A 24 hour urine Creatinine clearance >= 60 mL/min/1.73 m^2 - OR - A glomerular filtration rate (GFR) >= 60 mL/min/1.73 m^2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) - Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility - Serum glutamic-oxaloacetic transaminase (SGOT) aspartate aminotransferase [AST] or serum glutamate pyruvate transaminase (SGPT) aminotransferase [ALT] < 5 x upper limit of normal (ULN) for age - Total bilirubin < 2.5 mg/dL, unless attributable to Gilbert's Syndrome - Shortening fraction of >= 27% by echocardiogram or radionuclide scan (MUGA) - OR - Ejection fraction of >= 50% by echocardiogram or radionuclide scan (MUGA), choice of test according to local standard of care - Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and corrected carbon monoxide diffusing capability (DLCO) must all be >= 50% of predicted by pulmonary function tests (PFTs). - For children who are unable to perform for PFTs (e.g., due to age or developmental delay), the criteria are: no evidence of dyspnea at rest, oxygen (O2) saturation (Sat) > 92% on room air by pulse oximetry, not on supplemental O2 at rest, and not on supplemental O2 at rest - MPAL in first complete remission (CR1) for whom transplant is indicated. Examples include those patients who are poorly responsive to ALL therapy (end of induction failure( IF-MPAL) to ALL induction (see IF-MPAL note below), end of induction MRD = 5% or end-of-consolidation MRD > 0.01%), as well as patients treated with AML therapy - IF-MPAL: additional criterion for Induction failure for MPAL ONLY as per ALL1732: - An increasing number of circulating leukemia cells on 3 or more consecutive CBCs obtained at daily or longer intervals following day 8 of Induction therapy and prior to day 29 with confirmation by flow cytometry OR development of new sites of extramedullary disease, or other laboratory or clinical evidence of refractory disease or progression prior to the end of Induction evaluation (note that residual testicular disease at the end of Induction is an exception) - MPAL in > second complete remission (CR2) - ALL high-risk in CR1 for whom transplant is indicated. Examples include: induction failure, treatment failure as per minimal residual disease by flow cytometry > 0.01% after consolidation and not eligible for AALL1721 or AALL1721 not available/unwilling to enroll, hypodiploidy (< 44 chromosomes) with MRD+ > 0.01% after induction, persistent or recurrent cytogenetic or molecular evidence of disease during therapy requiring additional therapy after induction to achieve remission (e.g. persistent molecular BCR-ABL positivity), T cell ALL with persistent MRD > 0.01% after consolidation. - ALL in CR2 for whom transplant is indicated. Examples include: B-cell: early (=< 36 months from initiation of therapy) bone marrow (BM) relapse, late BM relapse (>= 36 months) with MRD >= 0.1% by flow cytometry after first re-induction therapy; T or B-cell: early (< 18 months) isolated extramedullary (IEM), late (>= 18 months) IEM, end-Block 1 MRD >= 0.1%; T-cell or Philadelphia chromosome positive (Ph+): BM relapse at any time - ALL in >= third complete remission (CR3) - Patients treated with chimeric antigen receptor T-cells (CART) cells for whom transplant is indicated. Examples include: transplant for consolidation of CART, loss of CART persistence and/or B cell aplasia < 6 months from infusion or have other evidence (e.g., MRD+) that transplant is indicated to prevent relapse - AML in CR1 for whom transplant is indicated. Examples include those deemed high risk for relapse as described in AAML1831: - FLT3/ITD+ with allelic ratio > 0.1 without bZIP CEBPA, NPM1 - FLT3/ITD+ with allelic ratio > 0.1 with concurrent bZIP CEBPA or NPM1 and with evidence of residual AML (MRD >= 0.05%) at end of Induction - Presence of RAM phenotype or unfavorable prognostic markers (other than FLT3/ITD) per cytogenetics, fluorescence in situ hybridization (FISH), next generation sequencing (NGS) results, regardless of favorable genetic markers, MRD status or FLT3/ITD mutation status - AML without favorable or unfavorable cytogenetic or molecular features but with evidence of residual AML (MRD >= 0.05%) at end of Induction - Presence of a non-ITD FLT3 activating mutation and positive MRD (>= 0.05%) at end of Induction 1 regardless of presence of favorable genetic markers. - AML in >= CR2 - MDS with < 5% blasts by morphology and flow cytometry (if available) on the pre-transplant bone marrow evaluation - Complete remission (CR) is defined as < 5% blasts by morphology and flow cytometry (if available) on the pre-transplant bone marrow evaluation with minimum sustained absolute neutrophil count (ANC) of 300 cells/microliter for 1 week or ANC > 500 cells/microliter. We will be collecting data from all approaches to MRD evaluation performed including NGS and polymerase chain reaction (PCR). It is strongly recommended that MPAL be evaluated using multidimensional flow cytometry and/or (KMT2Ar) qt PCR. It is strongly recommended that MPAL be evaluated using multidimensional flow cytometry and/or (KMT2Ar) qt PCR - DONOR ELIGIBILITY CRITERIA: - Matched Unrelated Donors: Unrelated donor candidates must be matched at high resolution at a minimum of 8/8 alleles (HLA-A, -B, -C, -DRB1). One-antigen HLA mismatches are not permitted. HLA matching of additional alleles is recommended according to National Marrow Donor Program (NMDP) guidelines, but will be at the discretion of local centers - Haploidentical Matched Family Members: - Minimum match level full haploidentical (at least 5/10; HLA-A, -B, -C, -DRB1, -DQB1 alleles). The following issues (in no particular order) should be considered in choosing a haploidentical donor: - Absent or low patient donor-specific antibodies (DSA) - Mean fluorescence intensity (MFI) of any anti-donor HLA antibody by solid phase immunoassay should be < 2000. Donors with higher levels are not eligible. - If a screening assay against pooled HLA antigens is used, positive results must be followed with specificity testing using a single antigen assay. The MFI must be < 2000 unless the laboratory has validated higher threshold values for reactivity for HLA antigens (such as HLA-C, -DQ, and -DP), that may be enhanced in concentration on the single antigen assays. Donor anti- recipient antibodies are of unknown clinical significance and do not need to be sent or reported. - Consult with Study Chair for the clinical significance of any recipient anti-donor HLA antibody. - If centers are unable to perform this type of testing, please contact the Study Chair to make arrangements for testing. - If killer immunoglobulin testing (KIR) is performed: KIR status by mismatch, KIR-B, or KIR content criteria can be used according to institutional guidelines. - ABO compatibility (in order of priority): - Compatible or minor ABO incompatibility - Major ABO incompatibility - CMV serostatus: - For a CMV seronegative recipient: the priority is to use a CMV seronegative donor when feasible - For a CMV seropositive recipient: the priority is to use a CMV seropositive donor when feasible - Age: younger donors including siblings/half-siblings, and second degree relatives (aunts, uncles, cousins) are recommended, even if < 18 years - Size and vascular access appropriate by center standard for peripheral blood stem cell (PBSC) collection if needed - Haploidentical matched family members: screened by center health screens and found to be eligible - Unrelated donors: meet eligibility criteria as defined by the NMDP or other unrelated donor registries. If the donor does not meet the registry eligibility criteria but an acceptable eligibility waiver is completed and signed per registry guidelines, the donor will be considered eligible for this study - Human immunodeficiency virus (HIV) negative - Not pregnant - MUD donors and post-transplant cyclophosphamide haplo donors should be asked to provide BM. If donors refuse and other donors are not available, PBSC is allowed. TCR-alpha beta/CD19 depleted haplo donors must agree to donate PBSC - Must give informed consent: - Haploidentical matched family members: Institution standard of care donor consent and Protocol-specific Donor Consent for Optional Studies - Unrelated donors: standard NMDP Unrelated Donor Consent Exclusion Criteria: - PATIENT EXCLUSION CRITERIA FOR ENROLLMENT: - Patients with genetic disorders (generally marrow failure syndromes) prone to secondary AML/ALL/MPAL with known poor outcomes because of sensitivity to alkylator therapy and/or TBI are not eligible (Fanconi Anemia, Kostmann Syndrome, Dyskeratosis Congenita, etc). Patients with Downs syndrome because of increased toxicity with intensive conditioning regimens. - Patients with any obvious contraindication to myeloablative HCT at the time of enrollment - Female patients who are pregnant are ineligible as many of the medications used in this protocol could be harmful to unborn children and infants - Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation - PATIENT EXCLUSION CRITERIA TO PROCEED TO HCT: - Patients with uncontrolled fungal, bacterial, viral, or parasitic infections are excluded. Patients with history of fungal disease during chemotherapy may proceed if they have a significant response to antifungal therapy with no or minimal evidence of disease remaining by computed tomography (CT) evaluation - Patients with active central nervous system (CNS) leukemia or any other active site of extramedullary disease at the time of initiation of the conditioning regimen are not permitted. - Note: Those with prior history of CNS or extramedullary disease, but with no active disease at the time of pre-transplant workup, are eligible - Pregnant or breastfeeding females are ineligible as many of the medications used in this protocol could be harmful to unborn children and infants |
Country | Name | City | State |
---|---|---|---|
Australia | The Children's Hospital at Westmead | Westmead | New South Wales |
Canada | Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec |
Canada | Hospital for Sick Children | Toronto | Ontario |
Canada | CancerCare Manitoba | Winnipeg | Manitoba |
United States | C S Mott Children's Hospital | Ann Arbor | Michigan |
United States | Children's Hospital Colorado | Aurora | Colorado |
United States | Johns Hopkins University/Sidney Kimmel Cancer Center | Baltimore | Maryland |
United States | Children's Hospital of Alabama | Birmingham | Alabama |
United States | Montefiore Medical Center - Moses Campus | Bronx | New York |
United States | Roswell Park Cancer Institute | Buffalo | New York |
United States | Medical University of South Carolina | Charleston | South Carolina |
United States | Lurie Children's Hospital-Chicago | Chicago | Illinois |
United States | Cleveland Clinic Foundation | Cleveland | Ohio |
United States | Medical City Dallas Hospital | Dallas | Texas |
United States | UT Southwestern/Simmons Cancer Center-Dallas | Dallas | Texas |
United States | Children's Hospital of Michigan | Detroit | Michigan |
United States | City of Hope Comprehensive Cancer Center | Duarte | California |
United States | Duke University Medical Center | Durham | North Carolina |
United States | Cook Children's Medical Center | Fort Worth | Texas |
United States | University of Florida Health Science Center - Gainesville | Gainesville | Florida |
United States | Helen DeVos Children's Hospital at Spectrum Health | Grand Rapids | Michigan |
United States | Hackensack University Medical Center | Hackensack | New Jersey |
United States | Penn State Children's Hospital | Hershey | Pennsylvania |
United States | Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center | Houston | Texas |
United States | Riley Hospital for Children | Indianapolis | Indiana |
United States | University of Iowa/Holden Comprehensive Cancer Center | Iowa City | Iowa |
United States | University of Mississippi Medical Center | Jackson | Mississippi |
United States | Nemours Children's Clinic-Jacksonville | Jacksonville | Florida |
United States | Children's Mercy Hospitals and Clinics | Kansas City | Missouri |
United States | Arkansas Children's Hospital | Little Rock | Arkansas |
United States | Loma Linda University Medical Center | Loma Linda | California |
United States | Norton Children's Hospital | Louisville | Kentucky |
United States | University of Wisconsin Carbone Cancer Center | Madison | Wisconsin |
United States | Nicklaus Children's Hospital | Miami | Florida |
United States | University of Miami Miller School of Medicine-Sylvester Cancer Center | Miami | Florida |
United States | The Children's Hospital at TriStar Centennial | Nashville | Tennessee |
United States | Vanderbilt University/Ingram Cancer Center | Nashville | Tennessee |
United States | Yale University | New Haven | Connecticut |
United States | The Steven and Alexandra Cohen Children's Medical Center of New York | New Hyde Park | New York |
United States | Children's Hospital New Orleans | New Orleans | Louisiana |
United States | Laura and Isaac Perlmutter Cancer Center at NYU Langone | New York | New York |
United States | UCSF Benioff Children's Hospital Oakland | Oakland | California |
United States | University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma |
United States | AdventHealth Orlando | Orlando | Florida |
United States | Lucile Packard Children's Hospital Stanford University | Palo Alto | California |
United States | Phoenix Childrens Hospital | Phoenix | Arizona |
United States | Virginia Commonwealth University/Massey Cancer Center | Richmond | Virginia |
United States | Mayo Clinic in Rochester | Rochester | Minnesota |
United States | University of Rochester | Rochester | New York |
United States | Washington University School of Medicine | Saint Louis | Missouri |
United States | Johns Hopkins All Children's Hospital | Saint Petersburg | Florida |
United States | Primary Children's Hospital | Salt Lake City | Utah |
United States | Methodist Children's Hospital of South Texas | San Antonio | Texas |
United States | UCSF Medical Center-Mission Bay | San Francisco | California |
United States | New York Medical College | Valhalla | New York |
United States | Alfred I duPont Hospital for Children | Wilmington | Delaware |
Lead Sponsor | Collaborator |
---|---|
Children's Oncology Group |
United States, Australia, Canada,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Neutrophil engraftment (defined as achieving a donor derived absolute neutrophil count (ANC) >= 500/uL for three consecutive measurements on different days) | We will estimate the cumulative incidences of neutrophil engraftment by Day 30 and Day 100 post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either the Haplo or MUD arm and who undergo HCT. | Day 30 and Day 100 post-transplant | |
Other | Platelet engraftment (defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count > 20,000/µL and > 50,000/µL with no platelet transfusions in the preceding seven days) | We will estimate cumulative incidences of platelet engraftment by Day 30 and Day 100 post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either the Haplo or MUD arms and who undergo HCT. | Day 30 and Day 100 post-transplant | |
Other | Primary and secondary graft failure | Primary (defined as lack of donor-derived neutrophil engraftment) and secondary graft failure (defined as initial donor-derived neutrophil engraftment followed by subsequent decline in ANC to < 500/µL with loss of donor chimerism to < 10% donor CD3 in peripheral blood or bone marrow). We will estimate cumulative incidences of primary and separately, secondary graft failure by Day 60 post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either Haplo or MUD arms and who undergo HCT. | 60 days post transplant | |
Other | Acute graft versus host disease (aGVHD) Grades II-IV and III-IV | We will estimate cumulative incidences of Grade II-IV aGVHD and separately, Grade III-IV aGVHD by Days 100 and 180 post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either Haplo or MUD arms and who undergo HCT. | Day 100 and Day 180 post-HCT | |
Other | Transplant related mortality (TRM) | We will estimate cumulative incidences of transplant-related mortality by 6-months, 1-year, and 2-years post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either Haplo or MUD arms and who undergo HCT. | 6-months, 1, and 2-year post-HCT | |
Other | Relapse | We will estimate cumulative incidences of relapse by 6-months, 1-year, and 2-years post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either Haplo or MUD arms. | 6-months, 1, and 2-years post-HCT | |
Other | Graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) (using the standard definition and two landmark definitions) | We will estimate cumulative incidences of GRFS events using all three definitions by 1-year, 18-months, and 2-years post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either Haplo or MUD arms and who undergo HCT. | 1-year, 18-months, and 2-years post-HCT | |
Other | Mild, moderate, and severe chronic graft versus host disease (cGVHD) | We will estimate cumulative incidences of mild, moderate, and severe cGvHD by 6-months, 1-year, and 2-years post-HCT and corresponding 95% confidence intervals among enrolled, eligible patients randomized to either Haplo or MUD arms and who undergo HCT. | 6-months, 1, and 2-year post-HCT | |
Other | Significant fungal infections (defined as proven or probable fungal infection according to the De-Pauw definition) | We will estimate the cumulative incidence of significant fungal infections by 1-year post-HCT and the corresponding 95% confidence interval among enrolled, eligible patients randomized to either Haplo or MUD arms. | Up-to 1-year post-HCT | |
Other | Viremia (with or without end organ disease) requiring hospitalization and/or systemic antiviral therapy | We will estimate the cumulative incidence of viremia by 1-year post-HCT and the corresponding 95% confidence interval among enrolled, eligible patients randomized to either Haplo or MUD arms. | Up to 1-year post-HCT | |
Other | Sinusoidal obstruction syndrome (SOS) (defined by the Cairo criteria and, separately, the EBMT, Baltimore, and modified Seattle criteria) | We will estimate the proportion of patients experiencing SOS under the separate definitions by 100-days post-HCT and the corresponding 95% confidence interval among enrolled, eligible patients randomized to either Haplo or MUD arms. | Up to 100-days post-HCT | |
Other | Transplant-associated thrombotic microangiopathy (TA-TMA) | We will estimate the proportion of TA-TMA events up-to 100-days post-HCT and the corresponding 95% confidence interval among enrolled, eligible patients randomized to either Haplo or MUD arms and who undergo HCT. | Up to 100-days post-HCT | |
Other | Reconstitution of T, B, and natural killer (NK) cells and immunoglobulins | We will estimate the median of the distribution of T, B, NK cells, and immunoglobulins among patients given each of three HCT methods (Haplo PTCy, Haplo alpha-beta T-cell depletion, and MUD). | At 30-days, 60-days, 100-days, 180-days and 365-days after HCT | |
Other | Vaccination-specific antibody titers | We will estimate theedian antibody titer among patients given each of three HCT methods (haplo PTCy, haplo alpha-beta T-cell depletion, and MUD). | 12-18 months post-HSCT | |
Other | Resource utilization (defined using the length of HCT hospital stay including readmissions within 100-days post-HCT, and using costs (inpatient) within the first 100-days and 2-years post HCT using Public Health Information System) | The average proportion of days spent hospitalized will be computed for patients randomized to the Haplo and MUD arms and corresponding 95% confidence intervals will be reported. The median inpatient PHIS adjusted costs by day 100 and 2-years post-HCT will be calculated for patients randomized to Haplo and MUD arms. | 100-days and 2-years post-HCT | |
Other | incorporating total body irradiation (TBI) delivered with volumetric modulated arc therapy (VMAT) or tomotherapy | Will be assessed by quality assurance monitoring. Will review treatment plans along with treatment received using data submitted to IROC and will describe the proportion of patients whose planned and actual TBI treatments align among those who received VMAT or tomotherapy. Will quantify the proportion of patients receiving VMAT or tomotherapy TBI who experience serious adverse events (defined as any AE reported through CTEP-AERS) and, separately, who experience any unexpected AE. Will estimate the cumulative incidence of death or relapse (DFS as a composite event) as well as death and relapse separately, and TRM by 0.5, 1, and 2-years year post-transplant from the time of starting TBI among those who receive VMAT or tomotherapy. For relapse and TRM, competing risk analysis will be used, as described under 9.3.5.1. 95% confidence intervals for each of these point estimates will also be provided. | Date of initiating TBI through 100 days post-transplant. | |
Primary | Severe GVHD (Grade III-IV acute GVHD or chronic GVHD requiring systemic immunosuppressive therapy) | We will estimate the cumulative incidence of severe GVHD at 1-year post-HCT and corresponding 95% confidence interval among enrolled and eligible patients randomly assigned to either HAPLO or MUD arms who actually undergo HCT. | Up-to 1-year post hematopoietic cell transplantation (HCT) | |
Primary | Disease free survival (DFS) (where an event is the occurrence of death from any cause or relapse) | We will estimate the cumulative incidence at 1 year post randomization and the corresponding 95% confidence interval among all enrolled and eligible patients randomly assigned to either HAPLO or MUD arms. | From date of randomization to up-to 1 year post randomization | |
Secondary | Overall survival (OS) | We will estimate the cumulative incidence of all-cause mortality events up-to 1-year post-HCT and the corresponding 95% confidence interval among all enrolled, eligible, randomized patients. | From date of randomization to up-to 1-year post-HCT | |
Secondary | Summary score from the Generic Pediatric Quality of Life Inventory (PedsQL) (excluding School Functioning) | For enrolled, eligible patients who consent to optional QoL studies, we will estimate the mean PedsQL score (excluding School Functioning) among patients randomized to the Haplo arm, the MUD arm, and among patients who enroll to arm C. For each, we will also calculate a corresponding 95% confidence interval. | At 6-months, 1 year and 2 years post-HCT | |
Secondary | Summary score from the PedsQL Stem Cell Transplant module | For enrolled, eligible patients who consent to optional QoL studies, we will estimate the mean PedsQL Stem Cell Transplant module score among patients randomized to the Haplo arm and the MUD arm, and among patients who enroll to arm C. For each, we will calculate a corresponding 95% confidence interval. | 6-months, 1 year and 2 years post-HCT |
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