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Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not been proved to be good for severe aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we reported successful double units UCBT after engraftment failure with single unit with promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus TBI conditioning regimen for double units cord blood transplantation was proposed for the patient with severe aplastic anemia without HLA-matched donor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Fludarabine | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cyclophosphosphamide, TBI | Other | fludarabine (30 mg/m2 once daily i.v. on days -7, -6, -5, -4, -3, & -2) (if body weight < 10 kg or if age < 1 year: 1.33 mg/kg) cyclophosphamide (60 mg/kg once daily i.v. on days -9, & -8) total body irradiation (3G on days -1) |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia. | From Oct 2008. to Sep 2011 |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the incidence and severity of toxicity and treatment related mortality | From Oct 2008. to Sep 2011 | |
| To evaluate overall and event free survival rate | From Oct 2008. to Sep 2011 | |
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Inclusion Criteria:
Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.
Peripheral blood
Bone marrow
No prior hematopoietic stem cell transplantation.
Age: no limits.
Performance status: ECOG 0-2.
Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.
Patients must lack any active viral infections or active fungal infection.
No appropriate donor
Appropriate cord blood is available: matched at least in 4/6 of A, B, DR loci.
Patients (or one of parents if patients age < 19) should sign informed consent.
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Hyoung Jin Kang, M.D, Ph.D | Contact | 82 2 2072 3304 | kanghj@snu.ac.kr | |
| Ji Won Lee, M.D | Contact | 82 2 2072 0177 | agnesjw@hanmail.net |
| Name | Affiliation | Role |
|---|---|---|
| Hyo Seop Ahn, M.D, Ph.D | The Korean Society of Pediatric Hematology Oncology | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Seoul National University Hospital | Recruiting | Seoul | 110-744 | South Korea |
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| To evaluate acute and chronic GVHD |
| From Oct 2008. to Sep 2011 |
| To evaluate immunologic recovery after CBT | From Oct 2008. to Sep 2011 |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| D000070642 | Brain Injuries, Traumatic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
| D001930 | Brain Injuries |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D006259 | Craniocerebral Trauma |
| D020196 | Trauma, Nervous System |
| D014947 | Wounds and Injuries |
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| ID | Term |
|---|---|
| D014916 | Whole-Body Irradiation |
| ID | Term |
|---|---|
| D011878 | Radiotherapy |
| D013812 | Therapeutics |
| D008919 | Investigative Techniques |
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