Donor Stem Cell Transplant After Chemotherapy for the Treatment of Recurrent or Refractory High-Risk Solid Tumors in Pediatric and Adolescent-Young Adults

  • End date
    May 9, 2025
  • participants needed
  • sponsor
    M.D. Anderson Cancer Center
Updated on 3 March 2021
stem cell transplantation
bone marrow procedure
glomerular filtration rate
tumor cells
cancer chemotherapy
solid tumor
stem cell transplant
primitive neuroectodermal tumor
neuroectodermal tumor
peripheral primitive neuroectodermal tumor
malignant peripheral nerve sheath tumor


This phase II trial investigates side effects and how well donor stem cell transplant after chemotherapy works in treating pediatric and adolescent-young adults with high-risk solid tumor that has come back (recurrent) or does not respond to treatment (refractory). Chemotherapy drugs, such as fludarabine, thiotepa, etoposide, melphalan, and rabbit anti-thymocyte globulin work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells.



I. To assess tolerability of allogeneic hematopoietic stem cell transplantation (HCT) for patients with chemo-responsive recurrent/refractory solid tumors as defined by transplant-related mortality (TRM) at day 30 and the rate of grade III or higher organ toxicity (Bearman Regimen-Related Toxicities Scale) attributable to conditioning occurring within 30 days.


I. Assess median time to platelet and neutrophil engraftment. II. Assess incidence of acute graft-versus-host disease (aGVHD) by day 100. III. Assess incidence of chronic GVHD (cGVHD) at day 100 and one year. IV. Assess rate of grade II organ toxicity through day 100. V. Assess rate of graft failure (primary and secondary) through day 100. VI. Assess rate of infectious complications through day 100. VII. Assess progression free survival (PFS) at day 100,180 and 365. VIII. Assess cumulative incidence of relapse, overall survival (OS) at 100 days and 1 year.


CONDITIONING REGIMEN: Patients receive thiotepa intravenously (IV) over 2-4 hours and etoposide IV over 60 minutes on days -8 to -6, melphalan IV over 20 minutes on days -5 and -4, and fludarabine phosphate IV over 1 hour on days -5 to -3 in the absence of disease progression or unacceptable toxicity. Patients receiving umbilical cord transplant also receive rabbit anti-thymocyte globulin IV on days -4 and -3.

TRANSPLANT: Patients undergo HSCT on day 0.

GVHD PROPHYLAXIS: Beginning day -2, patients receive tacrolimus or cyclosporine IV continuously until able to receive orally (PO). Patients continue tacrolimus or cyclosporine PO to day 60 and tapered to day 100. Patients also receive mycophenolate mofetil PO or IV every 8 hours until day 40 and tapered to day 90.

After completion of HSCT, patients are followed up for up to 1 year.

Condition Ewing's sarcoma, Desmoplastic Small Round Cell Tumor, Sarcoma, Sarcoma, Sarcoma (Pediatric), Soft Tissue Sarcoma, Recurrent Malignant Solid Neoplasm, Recurrent Neuroblastoma, Recurrent Rhabdomyosarcoma, Refractory Malignant Solid Neoplasm, Recurrent Malignant Peripheral Nerve Sheath Tumor, Sarcoma (Pediatric), Refractory Neuroblastoma, Refractory Rhabdomyosarcoma, Soft Tissue Sarcoma, Recurrent Desmoplastic Small Round Cell Tumor, Refractory Desmoplastic Small Round Cell Tumor, Refractory Malignant Peripheral Nerve Sheath Tumor
Treatment cyclosporine, fludarabine phosphate, mycophenolate mofetil, etoposide, melphalan, allogeneic hematopoietic stem cell transplantation, Tacrolimus, thiotepa, Lapine T-Lymphocyte Immune Globulin
Clinical Study IdentifierNCT04530487
SponsorM.D. Anderson Cancer Center
Last Modified on3 March 2021


Yes No Not Sure

Inclusion Criteria

Pathological criteria, including malignant recurrent/refractory solid tumors. This would include
Ewing's/peripheral primitive neuroectodermal tumor (PNET)
Malignant peripheral nerve sheath tumor, neurofibrosarcoma
Neuroblastoma (patients who are ineligible for tandem autologous transplant or who are at least 3 months post autologous HCT)
Desmoplastic small round cell tumor (DSRCT)- both new diagnoses as well as recurrent/refractory disease
Patients must have chemo-responsive disease, defined as; 30% or greater decrease in the tumor target lesions when compared to its pre-treatment evaluation. Patients with complete response will be eligible to participate
Available suitable HCT donor
Creatinine clearance or glomerular filtration rate (GFR) >= 50 ml/min/1.73m^2, and not requiring dialysis
Diffusing capacity of lung for carbon monoxide (DLCO) (corrected for hemoglobin) >= 50% predicted. If unable to perform pulmonary function tests, then oxygen (O2) saturation >= 92% in room air
Bilirubin =< 3 x upper limit of normal (ULN) and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =< 5 x for age (with the exception of isolated hyperbilirubinemia due to Gilbert's syndrome)
DONOR: Matched related donor bone marrow (10 of 10 HLA alleles [HLA-A, B, C, DR, and DQ]. Matched related donor peripheral blood stem cell (PBSC) is allowed only if collection of bone marrow (BM) is not available or refused by parents/donor
DONOR: Matched allogeneic umbilical cord blood (UCB): related
High-resolution matching at A,B, DRB1 (minimum 4/6)
KIR major histocompatibility complex (MHC) class 1 preferential mismatch (minimum 4/6)
DONOR: Matched allogeneic umbilical cord blood: unrelated
High-resolution matching at A,B, DRB1 (minimum 4/6) KIR MHC class 1 preferential mismatch (minimum 4/6)

Exclusion Criteria

Lack of histocompatible suitable related donor/ graft source
End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning regimen
Renal failure requiring dialysis
Congenital heart disease resulting in congestive heart failure
Ventilatory failure: requires invasive mechanical ventilation
Human immunodeficiency virus (HIV) infection
Uncontrolled bacterial, viral, or fungal infections (currently taking medication yet clinical symptoms progress); stable, controlled disease with treatment is not an exclusion criteria
A female of reproductive potential who is pregnant, planning to become pregnant during the study, or is nursing a child
Any patient who does not fulfill the inclusion criteria listed above
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