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A Phase II Study of Reduced Intensity Allogeneic Bone Marrow Transplant for the Treatment of Myelodysplastic Syndromes

Phase 2
18 Years
70 Years
Not Enrolling
Leukemia, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms

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Trial Information

A Phase II Study of Reduced Intensity Allogeneic Bone Marrow Transplant for the Treatment of Myelodysplastic Syndromes


- Determine the complete response rate in patients with myelodysplastic syndromes treated
with reduced-intensity allogeneic bone marrow transplantation, including photopheresis,
total body irradiation, and pentostatin.

- Determine the disease-free and overall survival of patients treated with this regimen.

- Determine the engraftment rate of donor cells in patients treated with this regimen.

- Determine the toxicity of this regimen in these patients.

- Determine the extent and duration of acute and chronic graft-versus-host disease in
patients treated with this regimen.

OUTLINE: This is a multicenter study.

- Preparative Regimen: Patients undergo photopheresis using methoxsalen on days -7 and -6
and receive pentostatin IV continuously on days -5 and -4. Total body irradiation is
administered on days -3 and -2 for a total of 3 doses.

- Transplantation: Allogeneic bone marrow or peripheral blood stem cells are infused on
day 0.

- Acute graft-vs-host-disease (GVHD) prophylaxis: Patients receive cyclosporine IV on
days -1 to 30 and then orally every 12 hours. Cyclosporine dose is then tapered
beginning after day 50 and continuing for 6 months in the absence of GVHD. Once
cyclosporine dose is significantly decreased, oral mycophenolate mofetil (MMF) is then
administered twice a day. MMF dose is then tapered for 12 months in the absence of
GVHD. Patients also receive methotrexate IV on days 1 and 3.

Patients are followed every 3 months for 2 years, every 6 months for 3 years, and then
annually thereafter.

PROJECTED ACCRUAL: A total of 33 patients will be accrued for this study within 2.1 years.

Inclusion Criteria


- One of the following cytologically proven myelodysplastic syndromes

- Refractory anemia (RA)

- RA with ringed sideroblasts

- RA with excess blasts

- Chronic myelomonocytic leukemia

- International Prognosis Scoring System (IPSS) score of at least 0.5 OR red cell
transfusion dependence for at least 6 months (2 units per month)

- Patients with an IPSS score less than 0.5 may be eligible provided they
previously had a higher IPSS score and received chemotherapy at that time

- Suitable HLA-matched donor (related or unrelated) available

- No cord blood donors

- Related donors must be genotypically matched (HLA A, B and DR) at 5/6 or 6/6
loci and may be a sibling, parent, or child

- Unrelated donors must have high resolution typing done at A, B, C and DR, and
must be matched at all or may have a single antigen or allele mismatch at no
more than one of these loci

- Patients must have < 20% blasts on bone marrow study within 1 month of study entry



- 18 to 70

Performance status

- ECOG 0-1

Life expectancy

- At least 6 months


- See Disease Characteristics

- Serum erythropoietin level greater than 100 for patients who have not received a
prior course of epoetin alfa

- No iron deficiency

- Iron deficiency anemia treated with iron replacement therapy allowed


- Bilirubin less than 2.0 mg/dL

- Alkaline phosphatase less than 2 times upper limit of normal (ULN)

- AST and ALT less than 3 times ULN


- Creatinine less than 2.0 mg/dL OR

- Creatinine clearance greater than 50 mL/min


- LVEF at least 45% by MUGA or echocardiogram


- DLCO at least 50% of predicted (corrected for hemoglobin)

- FEV_1 at least 50% of predicted


- Physically and psychologically capable of undergoing study regimen

- Able to receive 600 cGy of total body irradiation

- HIV negative

- Not pregnant or nursing

- Negative pregnancy test

- No other medical condition that would reduce life expectancy

- No active ongoing infection


Biologic therapy

- See Disease Characteristics

- See Hematopoietic

- At least 90 days since prior autologous bone marrow transplantation

- No prior myeloablative or nonmyeloablative allogeneic transplantation for
myelodysplastic syndrome or acute myeloid leukemia


- Recovered from prior chemotherapy

Endocrine therapy

- Not specified


- Not specified


- Not specified

Type of Study:


Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Complete response rate at day 100 after transplant

Safety Issue:


Principal Investigator

Selina M. Luger, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Abramson Cancer Center of the University of Pennsylvania


United States: Federal Government

Study ID:




Start Date:

May 2005

Completion Date:

Related Keywords:

  • Leukemia
  • Myelodysplastic Syndromes
  • Myelodysplastic/Myeloproliferative Neoplasms
  • chronic myelomonocytic leukemia
  • de novo myelodysplastic syndromes
  • previously treated myelodysplastic syndromes
  • refractory anemia
  • refractory anemia with excess blasts
  • refractory anemia with ringed sideroblasts
  • secondary myelodysplastic syndromes
  • atypical chronic myeloid leukemia, BCR-ABL1 negative
  • myelodysplastic/myeloproliferative neoplasm, unclassifiable
  • Neoplasms
  • Leukemia
  • Myelodysplastic Syndromes
  • Preleukemia
  • Myeloproliferative Disorders
  • Myelodysplastic-Myeloproliferative Diseases



Mayo Clinic Scottsdale Scottsdale, Arizona  85259
Mayo Clinic - Jacksonville Jacksonville, Florida  32224
Mayo Clinic Cancer Center Rochester, Minnesota  55905
Tufts-NEMC Cancer Center Boston, Massachusetts  02111
Jewish Hospital Cancer Center Cincinnati, Ohio  45236
Abramson Cancer Center of the University of Pennsylvania Philadelphia, Pennsylvania  19104-4283