Phase 3
N=10
Stem Cell Transplant for Bone Marrow Failure Syndromes
Diamond-Blackfan Anemia · Kostmann's Neutropenia · Shwachman-Diamond Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00176878 ↗Enrolled (actual)
10
Serious AEs
50.0%
Results posted
Aug 2009
Primary outcome: Primary: Number of Patients Alive (Survival) at 2 Years — 6 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Stem cell transplant (Procedure); Fludarabine monophosphate (Drug); Total lymphoid irradiation (Procedure); Busulfan (Drug); anti-thymocyte globulin (Biological)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Mar 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Patients Alive (Survival) at 2 Years |
6 | — |
| SECONDARY Number of Patients Alive at Three Years (Survival) |
6 | — |
| SECONDARY Number of Patients With Succcessful Engraftment After Transplantation |
10 | — |
| SECONDARY Number of Patients With Grade 2-4 Acute Graft Versus Host Disease |
5 | — |
| SECONDARY Number of Patients With Chronic Graft Versus Host Disease |
3 | — |
| SECONDARY Number of Patients With Disease Recurrence |
— | — |
Summary
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.
Eligibility Criteria
Inclusion Criteria
- Patients eligible for transplantation under this protocol will be 6 transfusions yearly despite steroid therapy.
- Evidence of developing aplasia or myelodysplasia will also be criteria for transplantation.
- Kostmann's Neutropenia, Shwachman-Diamond syndrome:
- Patients must have been previously diagnosed as having a clinical picture characteristic of Shwachman-Diamond syndrome (exocrine pancreatic insufficiency, growth retardation, metaphyseal dysostosis, neutropenia), or must have a bone marrow aspirate consistent with Kostmann's neutropenia, with no evidence of acute leukemia.
- Patients must have failed therapy with granulocyte-colony stimulating factor (G-CSF), as determined by an inability to maintain an absolute neutrophil count (ANC) >750 cells/ml(3), or manifesting recurrent infections despite G-CSF administration resulting in life threatening infections or repeated hospitalizations ( 35 years of age
- Karnofsky score 3.0, ALT >150, or active hepatitis
- Pulmonary function tests with forced volume vital capacity (FVC) and forced expiratory volume (FEV) <70%; O2 saturation <94%
- Renal dysfunction with glomerular filtration rate (GFR) <30% of predicted.
- Cardiac compromise, with left ejection fraction <45%.
- Severe, stable neurologic impairment.
- Human immunodeficiency virus (HIV) positivity.
- Pregnant or lactating females
Data sourced from ClinicalTrials.gov (NCT00176878). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.