Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

  • STATUS
    Recruiting
  • days left to enroll
    86
  • participants needed
    20
  • sponsor
    Masonic Cancer Center, University of Minnesota
Updated on 22 March 2022
cancer
stem cell transplantation
myeloid leukemia
lymphoid leukemia
fludarabine
anemia
mycophenolate mofetil
blood stem cell transplant
myelodysplastic syndromes
busulfan
cyclosporine
acute leukemia
myelodysplasia
white blood cell count
umbilical cord blood transplantation
flow cytometry
filgrastim
hypodiploidy
ejection fraction
granulocyte colony stimulating factor
melphalan
cell transplantation
leukemia
bone marrow procedure
lymphocytic leukemia
minimal residual disease
blast cells
colony stimulating factor
combination chemotherapy
white blood cells
PCR test
wbc count
raeb
nucleated cells

Summary

RATIONALE: Giving chemotherapy, such as busulfan, fludarabine, and melphalan, before a donor umbilical cord blood stem cell transplant helps stop the growth of abnormal or cancer cells and prepares the patient's bone marrow for the stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil may stop this from happening.

PURPOSE: This phase II trial is studying how well combination chemotherapy followed by a donor umbilical cord blood transplant works in treating infants with high-risk acute leukemia or myelodysplastic syndromes.

Description

OBJECTIVES

Primary

  • Determine the incidence of engraftment, defined as achieving donor-derived neutrophil count > 500/mm³ by day 42, in infants with high-risk acute myeloid leukemia, acute lymphoblastic leukemia, or myelodysplastic syndromes treated with a non-irradiation containing myeloablative conditioning regimen comprising busulfan, fludarabine, and melphalan followed by double umbilical cord blood transplantation (UCBT) with two partially HLA-matched units.

Secondary Objectives

  • Determine the incidence of transplant-related mortality (TRM) at 6 months after UCBT
  • Evaluate pattern of chimerism after double UCBT
  • Determine the incidence of platelet engraftment at 1 year after UCBT
  • Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV at day 100 after UCBT
  • Evaluate the developmental outcome after UCBT

Transplant Related Objectives

  • Determine the incidence of chronic GVHD at 1 year after UCBT
  • Determine the survival and disease free survival at 1 and 2 years after UCBT
  • Determine the incidence relapse at 1 and 2 years after UCBT

Details
Condition 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, Refractory Anemia, De Novo Myelodysplastic Syndrome, Childhood Myelodysplastic Syndrome
Treatment cyclosporine, fludarabine phosphate, mycophenolate mofetil, filgrastim, busulfan, melphalan, Umbilical Cord Blood Transplantation
Clinical Study IdentifierNCT00357565
SponsorMasonic Cancer Center, University of Minnesota
Last Modified on22 March 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

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

Exclusion Criteria

Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
History of HIV infection or known positive serology
Myeloablative transplant within the last 6 months
Evidence of active extramedullary disease (including central nervous system leukemia)
Clear my responses

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