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Patients 18 years old or younger with marrow aplasia or single lineage cytopenias (Arm A) will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.6-0.8 mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).

Patients 18 years old or younger with MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.8-1.0mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).

Patients 19 years old or older with marrow aplasia or MDS or AML will be will be conditioned for transplantation with intravenous busulfan (busulfex®) (0.4mg/Kg/dose q 12 hours x 4 doses), cyclophosphamide (10 mg/Kg/dose x 4 doses) and fludarabine (35mg/m2/day x 4 doses).

A standard dose of busulfan, associated with excellent outcomes in our previous trial will be used for young patients with marrow aplasia (arm A).

A higher dose of busulfan will be used in younger patients with MDS and AML (arm B) to maximize disease control.

A lower dose of busulfan will be used in older patients (arm C) to minimize toxicity.

Arms A, B and C - Fludarabine will be given IV over 30 minutes daily for 4 days. The dose will be adjusted according to renal function according to Institutional guidelines.

Arms A, B and C - Cytoxan will be given as a 1-2 hour infusion for 4 days. The dose will be adjusted according to patients ideal body weight for obese patients.

Arms A, B and C - 4 doses will be given prior to transplant to promote engraftment

Anti-Thymocyte Globulin (Rabbit)

The source of stem cells for all patients will be peripheral blood stem cells (PBSC) induced and mobilized by treatment of the donor with G-CSF for 4-6 days. T-cell depletion will be uniformly performed by positive CD34 selection with the use of the Miltenyi system (CliniMACS device)

The CliniMACS device

All patients will also receive G-CSF post-transplant to foster engraftment.

G-CSF

Farid Boulad, MD

The goal of this study is to see if the study therapy can decrease the chemotherapy-related side effects while maximizing the effectiveness of disease control. The physicians will also be studying the effect of removing T-cells from the donor"s stem cells before transplant. T-cells are a type of white blood cell that may help cause a serious side effect of transplant called Graft versus Host Disease (GVHD). The way it removes the T-cells from the donor stem cells is actually by selecting only the stem cells (called CD34 cells) by using a device called CliniMACS. This process is called CD34 selection. The CliniMACS® device is currently under the supervision of the FDA .

A three-arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and ATG

A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia

A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

Primary non-engraftment is diagnosed when the patient fails to achieve an ANC ≥500/µl at any time in the first 28 days post-transplant. If (1) after achievement of an ANC ≥500/mm\^3, the ANC declines to \<500/mm\^3 for more than 3 consecutive days in the absence of relapse, or, (2) there is absence of donor cells in the marrow and/or blood as demonstrated by chimerism assay in the absence of relapse, a diagnosis of secondary graft failure is made. The patient is not evaluable for graft failure or rejection if recurrence of host MDS is detected concurrently.

Pediatric Brain Tumor Consortium

Memorial Sloan Kettering Cancer Center

All-Cause Mortality, Serious, or Other (non-serious) Adverse Events were monitored/assessed in the "Intermediate Risk Patients" Arm/Group but none observed.

Good Risk Patients

Intermediate Risk Patients

High Risk Patients

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Dr. Farid Boulad, MD

Data were not collected. No participants received treatment and study terminated due to slow accrual

Graft Failure or Rejection

Overall Study

Hematologic Neoplasms

Melphalan

1. Cyclophosphamide and Total Body Irradiation (CY-TBI)-based regimens that include at least 120 mg/kg cyclophosphamide and at least 1200 cGy of fractionated TBI.
2. Busulfan and cyclophosphamide (BU-CY)-based regimens that include at least 14 mg/kg busulfan orally or 11.2 mg/kg busulfan intravenously (14 x 0.8 correction factor) or a targeted busulfan dosing strategy aimed at a serum concentration greater than 600 ng/mL at steady state and at least 120 mg/kg cyclophosphamide.
3. Fludarabine and melphalan (Flu-Mel)-based regimens that include a fludarabine dose of least 120 mg per m2 and a melphalan dose of at least 140 mg per m2.
4. Fludarabine, busulfan, and ATG (Flu-Bu-ATG)-based regimens that include a fludarabine dose of at least 120 mg/m2, at least 8 mg/kg busulfan orally or 250 mg/m2 busulfan intravenously, and at least 40 mg/kg equine ATG or 4 mg/kg rabbit ATG. Institutional standards should be followed for targeting plasma levels.

Blood stem cell versus bone marrow transplant

Shakila P. Khan, M.D.

This study will compare the outcomes of study participants who have had peripheral blood stem cell transplantation or with those who have had bone marrow transplantation from an unrelated donor for the treatment of serious hematological malignancies.

This study is a randomized, open-label, multicenter, prospective, comparative trial of granulocyte colony stimulating factor (G-CSF) mobilized peripheral blood stem cells verses marrow from unrelated donors for transplantation in patients with hematologic malignancies. The study will be comparing two-year survival probabilities between process has many steps after a matched donor is found. The donor also needs to give permission to join this study. Participants will be randomized to either peripheral blood stem cells (PBSC) or bone marrow. The kind of transplant the participant receives is random. Neither the participant nor the doctor may choose the type of transplant.

Participants will receive vaccinations for diphtheria, tetanus, hepatitis B and pneumococcus. Blood samples will be drawn as part of a participant's normal care. Blood samples will also be collected to see if infection fighting cells are working. These samples will be collected at 12 weeks, one year and then yearly until three years post transplant. Information will be collected from the participant's medical history. Participants will also have phone interviews conducted and questions will be asked about their physical and emotional health. Participants taking part in this study may be followed for up to 5 years.

Comparison of Peripheral Blood Stem Cell Transplantation With Bone Marrow Transplantation for the Treatment of Serious Hematological Malignancies

A Phase III Randomized, Multicenter Trial Comparing G-CSF Mobilized Peripheral Blood Stem Cell With Marrow Transplantation From HLA Compatible Unrelated Donors.

The primary objective is to compare two-year survival probabilities between patients in the two study arms using an intent-to-treat analysis

Transplanted patients randomized to the two study arms will be compared for the following endpoints: survival, incidences of neutrophil and platelet engraftment, graft failure, acute graft-versus-host disease (GVHD), chronic GVHD, time off all immunosuppressive therapy, relapse, infections, adverse events, immune reconstitution, and quality of life. Donors in each arm of the study will be compared for time to return to baseline toxicity score, CBC and WBC differential values after donation and quality of life.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia	NCT03600909	Fanconi Anemia Myelodysplastic... Acute Myelogeno...	Busulfan Fludarabine Cyclophosphamid... Anti-Thymocyte ... The CliniMACS d... G-CSF	1 Month -	Memorial Sloan Kettering Cancer Center
Comparison of Peripheral Blood Stem Cell Transplantation With Bone Marrow Transplantation for the Treatment of Serious Hematological Malignancies	NCT00275678	Blood Stem Cell...	Blood stem cell...	18 Years - 66 Years	Mayo Clinic

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