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Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders

Phase 2
55 Years
Not Enrolling
Leukemia, Lymphoma, Bone Marrow Failure Syndromes, Immunodeficiencies, Histiocytosis

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Trial Information

Unrelated Donor Stem Cell Transplant for Patients With Malignant and Non-Malignant Disorders

This is a non-randomized study to determine the tolerability and degree of engraftment of
unrelated matched donor allogeneic stem cell transplantation with either myeloablative or
reduced intensity conditioning in patients with selected malignant and non malignant
disorders. Patients will receive one of either full intensity or reduced intensity regimen
based on the patient's disease status, organ function and performance and determined by the

Inclusion Criteria:

- Adequate renal function defined as: serum creatinine 2.0 x normal, or creatinine
clearance or radioisotope GFR > 40 ml/min/m2 or > 40 ml/min/1.73 m2 or an equivalent
GFR as determined by the institutional normal range.

- Adequate liver function defined as: total bilirubin < 2.5 x normal; or SGOT (AST) or
SGPT (ALT) < 5.0 x normal.

- Adequate cardiac function defined as: shortening fraction of > 25% by echocardiogram,
or ejection fraction of > 40% by radionuclide angiogram or echocardiogram.

- Adequate pulmonary function defined as: DLCO > 35% by pulmonary function test. For
children who are uncooperative, no evidence of dyspnea at rest, no exercise
intolerance, and a pulse oximetry > 94% in room air.

- Diseases:

- CML (CP, AP or BC)



- Lymphoma (Hodgkin's and non-Hodgkin's)

- Non-malignant disorders

- Bone Marrow Failure Syndromes: Patients with the following diagnoses are eligible:

- Severe Aplastic Anemia:

- Fanconi Anemia

- Severe Congenital Neutropenia (Kostmann's Syndrome)

- Amegakaryocytic Thrombocytopenia

- Diamond-Blackfan Anemia

- Infantile Osteopetrosis

- Schwachman-Diamond Syndrome

- Dyskeratosis Congenita

- Other bone marrow failure syndromes at discretion of Principal Investigator

- Immunodeficiencies:

- SCIDS, all subtypes

- Combined Immunodeficiency Syndrome

- Wiskott-Aldrich syndrome

- Chronic Granulomatous Disease

- Chediak-Higashi Syndrome

- Leukocyte Adhesion Deficiency

- Other immunodeficiencies at discretion of Principal Investigator

- Inborn Errors of Metabolism (IEOM):

- Transplant is recommended for the following disorders: Hurler syndrome
(L-iduronidase deficiency, MPS-I), Maroteaux-Lamy syndrome
(galactosamine-4-sulfatase deficiency, MP VI), Sly syndrome (glucuronidase
deficiency, MPS-VII), Globoid cell Leukodystrophy
(galactocerebrosidasedeficiency), Metachromatic leukodystrophy (arylsulfatase A
deficiency), Childhood-onset X-linked adrenoleukodystrophy (X-ALD), Fucosidosis
(fucosidase deficiency), Mannosidosis, Aspartylglucosaminuria, Niemann-Pick
Disease Type B (acid sphingomyelinase deficiency), Gaucher disease
(glucocerebrosidase deficiency) Type I (non neuropathic), Other diagnoses may be
considered at the discretion of the Principal Investigator

- For X-ALD patients greater than 5 years of age, IQ > 80 is required. For other
patients greater than 5 years of age, IQ > 70 is required.

- For patients less than 5 years of age, the developmental quotient or clinical
neurodevelopmental examination should demonstrate potential for stabilization at
a level of functioning where continuous life support (e.g. mechanical
ventilation) would not be predicted to be required in the year following

- Histiocytosis:

- Hemophagocytic Lymphohistiocytosis (HLH)

- Familial Erythrophagocytic Lymphohistiocytosis

- Langerhans Cell Histiocytosis

- Malignant Histiocytosis

- Other Malignant and non-malignant diseases: Other malignant and non-malignant
diseases not listed above may be eligible if deemed appropriate by the Principal

Exclusion Criteria:

- Women who are pregnant and/or breast feeding are ineligible

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the safety and toxicity of myeloablative therapy (TBI + Melphalan) and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.

Outcome Time Frame:

Up to 10 years from start of study

Safety Issue:


Principal Investigator

Mitchell S Cairo, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Columbia University


United States: Institutional Review Board

Study ID:




Start Date:

November 2002

Completion Date:

April 2011

Related Keywords:

  • Leukemia
  • Lymphoma
  • Bone Marrow Failure Syndromes
  • Immunodeficiencies
  • Histiocytosis
  • Allogeneic Stem Cell Transplant
  • Unrelated donor
  • Cord blood donor
  • Histiocytosis
  • Immunologic Deficiency Syndromes
  • Leukemia
  • Lymphoma
  • Pancytopenia
  • Hemoglobinuria, Paroxysmal



Columbia University Medical CenterNew York, New York  10032