Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

Phase 2

Active, not recruiting

• Conditions: Leukemia; Myelodysplastic Syndromes; Childhood Acute Myeloid Leukemia in Remission; Recurrent Childhood Acute Myeloid Leukemia; Secondary Acute Myeloid Leukemia; Childhood Acute Lymphoblastic Leukemia in Remission; Previously Treated Myelodysplastic Syndrome; Secondary Myelodysplastic Syndrome; Refractory Anemia With Excess Blasts in Transformation; Refractory Anemia With Excess Blasts

• Registration Number: NCT00357565
• Lead Sponsor: Masonic Cancer Center, University of Minnesota

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2006-7-26
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Active, not recruiting
• Sex: All
• Target Recruitment: 33

Inclusion Criteria

Inclusion Criteria:<br><br> - Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched<br> single unit based on the following priority:<br><br> - 1st priority: 4/6 matched unit, cell dose >5 x 10-7 nucleated cells/kg<br><br> - 2nd priority: 5/6 matched unit, cell dose > 4 x 10-7 nucleated cells/kg<br><br> - 3rd priority: 6/6 matched unit, cell dose > 3 x 10-7 nucleated cells/kg<br><br> - Patients aged = 3 years at diagnosis (not age of transplant) with hematological<br> malignancy as detailed below:<br><br> - Acute myeloid leukemia: high risk CR1 as evidenced by:<br><br> - High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5,<br> 7, or 19 abnormalities; complex karyotype (>5 distinct changes); = 2<br> cycles to obtain complete response (CR); CR2 or higher; Preceding<br> myelodysplastic syndrome (MDS); All patients must be in CR or early<br> relapse (i.e., <15% blasts in BM).<br><br> - Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk<br> cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1<br> cycle to obtain CR; CR2 or higher; All patients must be in CR as defined<br> by hematological recovery, AND <5% blasts by light microscopy within the<br> bone marrow with a cellularity of =15%.<br><br> - Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory<br> anemia with severe pancytopenia or high risk cytogenetics. Blasts must be < 10%<br> by a representative bone marrow aspirate morphology.<br><br> - Persistent or rising minimal residual disease (MRD) after standard chemotherapy<br> regimens: Patients with evidence of minimal residual disease at the completion<br> of therapy or evidence of rising MRD while on therapy. MRD will be defined by<br> either flow cytometry (>0.1% residual cells in the blast gate with immune<br> phenotype of original leukemic clone), by molecular techniques (PCR or FISH) or<br> conventional cytogenetics (g-banding).<br><br> - New Leukemia Subtypes: A major effort in the field of pediatric hematology is<br> to identify patients who are of high risk for treatment failure so that<br> patients can be appropriately stratified to either more (or less) intensive<br> therapy. This effort is continually ongoing and retrospective studies identify<br> new disease features or characteristics that are associated with treatment<br> outcomes. Therefore, if new high risk features are identified after the writing<br> of this protocol, patients can be enrolled with the approval of two members of<br> the study committee.<br><br> - Recipients must have a Lansky score = 50% and have acceptable organ function defined<br> as:<br><br> - Renal: glomerial filtration rate > 60ml/min/1.73m^2<br><br> - Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal,<br><br> - Pulmonary function: oxygen saturation >92%<br><br> - Cardiac: left ventricular ejection fraction > 45%.<br><br> - Voluntary written informed consent before performance of any study-related procedure<br> not part of normal medical care.<br><br>Exclusion Criteria:<br><br> - Active infection at time of transplantation (including active infection with<br> Aspergillus or other mold within 30 days).<br><br> - History of HIV infection or known positive serology<br><br> - Myeloablative transplant within the last 6 months.<br><br> - Evidence of active extramedullary disease (including central nervous system<br> leukemia).

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Incidence of Engraftment

Secondary Outcome Measures

Name	Time	Method
Incidence of transplant-related mortality (TRM);Incidence of platelet engraftment;Incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV;Incidence of chronic graft-versus-host disease (GVHD);Incidence of relapse;Overall survival;Developmental Outcomes;Disease-free survival