Refractory anemia with excess blasts in transformation
diseaseOn this page
Also known as RAEB-T
Summary
Refractory anemia with excess blasts in transformation (MONDO:0015692) is a disease and 22 clinical trials. Top therapeutic interventions include fludarabine phosphate, cyclophosphamide anhydrous, and idarubicin. A subtype of refractory hematologic cancer — broader associated-gene and molecular evidence is on the parent page (see Disease family below).
At a glance
- Prevalence: <1 / 1 000 000 (Europe) [Orphanet-validated]
- Clinical trials: 22
Clinical features
Epidemiology
Prevalence records
1 prevalence record(s), Orphanet:
| Type | Class | Value | Geography | Validation |
|---|---|---|---|---|
| Annual incidence | <1 / 1 000 000 | 0.04 | Europe | Validated |
Identifiers
Disease identifiers
| Field | Value |
|---|---|
| Canonical name | refractory anemia with excess blasts in transformation |
| Mondo ID | MONDO:0015692 |
| Orphanet | 168960 |
| NCIT | C27080 |
| SNOMED CT | 110000005 |
| UMLS | C0280028 |
| MedGen | 124692 |
| GARD | 0020108 |
| MedDRA | 10038271 |
| Is cancer (heuristic) | no |
Also known as: RAEB-T · RAEB-t
Disease family
This is a subtype of refractory hematologic cancer. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.
Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › refractory hematologic cancer › refractory anemia with excess blasts in transformation
Related subtypes (5): refractory hairy cell leukemia, refractory precursor T-lymphoblastic lymphoma/leukemia, refractory plasma cell neoplasm, myelodysplastic syndrome with multilineage dysplasia, refractory cytopenia of childhood
Genetics & variants
GWAS landscape
No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.
Variant details and genetic-evidence tiers
No tiered GWAS variants or ClinVar records for this disease.
Genes & proteins
No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).
Function
No pathway enrichment — requires an associated-gene cohort.
Therapeutics
No druggable-target or therapeutic data for this disease’s cohort.
Clinical trials & evidence
Clinical trials
Clinical trials: 22.
Phase distribution (across all retrieved trials)
| Phase | Trials |
|---|---|
| PHASE1 | 11 |
| PHASE2 | 5 |
| PHASE1/PHASE2 | 4 |
| PHASE3 | 1 |
| Not specified | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00002798 | PHASE3 | COMPLETED | Combination Chemotherapy With or Without Bone Marrow Transplantation in Treating Children With Acute Myelogenous Leukemia or Myelodysplastic Syndrome |
| NCT00004918 | PHASE1/PHASE2 | COMPLETED | Vaccine Therapy Plus Immune Adjuvant in Treating Patients With Chronic Myeloid Leukemia, Acute Myeloid Leukemia, or Myelodysplastic Syndrome |
| NCT00005942 | PHASE1/PHASE2 | COMPLETED | Liposomal Daunorubicin and SU5416 in Treating Patients With Hematologic Cancer That Has Not Responded to Initial Therapy |
| NCT00015990 | PHASE2 | COMPLETED | Thalidomide in Treating Patients With Myelodysplastic Syndrome |
| NCT00052520 | PHASE1/PHASE2 | COMPLETED | Biological Therapy in Treating Patients With Advanced Myelodysplastic Syndrome, Acute or Chronic Myeloid Leukemia, or Acute Lymphoblastic Leukemia Who Are Undergoing Stem Cell Transplantation |
| NCT00084916 | PHASE2 | COMPLETED | CCI-779 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Myelodysplastic Syndromes, or Chronic Myelogenous Leukemia in Blastic Phase |
| NCT00096122 | PHASE1/PHASE2 | COMPLETED | Idarubicin, Cytarabine, and Tipifarnib in Treating Patients With Newly Diagnosed Myelodysplastic Syndromes or Acute Myeloid Leukemia |
| NCT00119366 | PHASE2 | TERMINATED | Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Total Body Irradiation, and Donor Stem Cell Transplant Followed by Cyclosporine and Mycophenolate Mofetil in Treating Patients With Advanced Acute Myeloid Leukemia or Myelodysplastic Syndrome |
| NCT00357565 | PHASE2 | COMPLETED | Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia |
| NCT01175785 | PHASE2 | COMPLETED | Infusion of Off-the-Shelf Expanded Cord Blood Cells to Augment Cord Blood Transplant in Patients With Hematologic Malignancies |
| NCT00005064 | PHASE1 | COMPLETED | PS-341 in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myeloid Leukemia in Blast Phase, or Myelodysplastic Syndrome |
| NCT00005845 | PHASE1 | COMPLETED | Tipifarnib in Treating Patients With Myelodysplastic Syndromes |
| NCT00006213 | PHASE1 | COMPLETED | BMS-214662 in Treating Patients With Acute Leukemia, Myelodysplastic Syndrome, or Chronic Myeloid Leukemia |
| NCT00008177 | PHASE1 | COMPLETED | Radiolabeled Monoclonal Antibody Therapy, Fludarabine Phosphate, and Low-Dose Total-Body Irradiation Followed by Donor Stem Cell Transplant and Immunosuppression Therapy in Treating Older Patients With Advanced Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndromes |
| NCT00087204 | PHASE1 | COMPLETED | Rebeccamycin Analog in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia, or Chronic Myelogenous Leukemia |
| NCT00095797 | PHASE1 | COMPLETED | XK469R in Treating Patients With Refractory Hematologic Cancer |
| NCT00098423 | PHASE1 | COMPLETED | Tanespimycin and Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Chronic Myelogenous Leukemia, Chronic Myelomonocytic Leukemia, or Myelodysplastic Syndromes |
| NCT00098826 | PHASE1 | COMPLETED | SB-715992 in Treating Patients With Acute Leukemia, Chronic Myelogenous Leukemia, or Advanced Myelodysplastic Syndromes |
| NCT00343798 | PHASE1 | COMPLETED | A Pilot Study to Evaluate the Co-Infusion of Ex Vivo Expanded Cord Blood Cells With an Unmanipulated Cord Blood Unit in Patients Undergoing Cord Blood Transplant for Hematologic Malignancies |
| NCT01834248 | PHASE1 | COMPLETED | DEC-205/NY-ESO-1 Fusion Protein CDX-1401and Decitabine in Treating Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia |
| NCT02553941 | PHASE1 | COMPLETED | Ibrutinib and Azacitidine for Treatment of Higher Risk Myelodysplastic Syndrome |
| NCT01146210 | Not specified | COMPLETED | Identification of de Novo Fanconi Anemia in Younger Patients With Newly Diagnosed Acute Myeloid Leukemia |
Drugs tested across these trials (top 30)
| Molecule | Max phase | Trials referencing |
|---|---|---|
| FLUDARABINE PHOSPHATE | 4 | 6 |
| CYCLOPHOSPHAMIDE ANHYDROUS | 4 | 3 |
| IDARUBICIN | 4 | 2 |
| ALDESLEUKIN | 4 | 1 |
| ASPARAGINASE | 4 | 1 |
| BUSULFAN | 4 | 1 |
| CYTARABINE | 4 | 1 |
| DAUNORUBICIN HYDROCHLORIDE | 4 | 1 |
| ETOPOSIDE | 4 | 1 |
| FILGRASTIM | 4 | 1 |
| HYDROCORTISONE | 4 | 1 |
| SARGRAMOSTIM | 4 | 1 |
| TEMSIROLIMUS | 4 | 1 |
| THIOGUANINE | 4 | 1 |
| TIPIFARNIB | 3 | 2 |
| BECATECARIN | 3 | 1 |
| INCOMPLETE FREUND’S ADJUVANT | 3 | 1 |
| PR1 LEUKEMIA PEPTIDE VACCINE | 3 | 1 |
| SEMAXANIB | 3 | 1 |
| TANESPIMYCIN | 3 | 1 |
| HILTONOL | 2 | 1 |
| ISPINESIB | 2 | 1 |
| BMS-214662 | 1 | 1 |
| R(+)XK469 | 1 | 1 |
| CHEMBL4786163 | 0 | 1 |
| CHEMBL2326523 | 0 | 1 |
Related Atlas pages
- Drugs: Fludarabine Phosphate, Cyclophosphamide, Idarubicin, Aldesleukin, Asparaginase, Busulfan, Cytarabine, Daunorubicin, Etoposide, Filgrastim, Hydrocortisone, Sargramostim, Temsirolimus, Thioguanine, Tipifarnib, Becatecarin, Incomplete Freund’S Adjuvant, PR1 LEUKEMIA PEPTIDE VACCINE, Semaxanib, Tanespimycin