busulfan has been researched along with Bare Lymphocyte Syndrome in 22 studies
| Excerpt | Relevance | Reference |
|---|---|---|
| "The pharmacokinetics of low-dose busulfan (BU) were investigated as a nonmyeloablative conditioning regimen for autologous gene therapy (GT) in pediatric subjects with adenosine deaminase-deficient severe combined immunodeficiency disease (ADA SCID)." | 7.96 | Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy. ( Ansari, M; Bradford, KL; Candotti, F; Garabedian, E; Gaspar, HB; Kohn, DB; Krajinovic, M; Liu, S; Shaw, KL; Tse, J; Wang, X, 2020) |
| "The pharmacokinetics of low-dose busulfan (BU) were investigated as a nonmyeloablative conditioning regimen for autologous gene therapy (GT) in pediatric subjects with adenosine deaminase-deficient severe combined immunodeficiency disease (ADA SCID)." | 3.96 | Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy. ( Ansari, M; Bradford, KL; Candotti, F; Garabedian, E; Gaspar, HB; Kohn, DB; Krajinovic, M; Liu, S; Shaw, KL; Tse, J; Wang, X, 2020) |
| "Human Severe Combined Immunodeficiency (SCID) is a prenatal disorder of T lymphocyte development that depends on the expression of numerous genes." | 1.39 | Tooth developmental anomalies in severe combined immunodeficiency disease and juvenile myelomonocytic leukemia: common clinical features and treatment outcomes. ( Angiero, F; Benedicenti, S; Cossellu, G; Farronato, G; Olivi, G; Seramondi, R, 2013) |
| "Busulfan doses were decreased in 69% of patients compared to conventional doses." | 1.31 | Improved clinical outcome of paediatric bone marrow recipients using a test dose and Bayesian pharmacokinetic individualization of busulfan dosage regimens. ( Aulagner, G; Bertrand, Y; Bleyzac, N; Dai, Q; Galambrun, C; Janoly, A; Jelliffe, RW; Magron, P; Maire, P; Martin, P; Souillet, G, 2001) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 3 (13.64) | 18.7374 |
| 1990's | 2 (9.09) | 18.2507 |
| 2000's | 5 (22.73) | 29.6817 |
| 2010's | 9 (40.91) | 24.3611 |
| 2020's | 3 (13.64) | 2.80 |
| Authors | Studies |
|---|---|
| White, SL | 1 |
| Lee, TD | 1 |
| Toy, T | 1 |
| Carroll, JE | 1 |
| Polsky, L | 1 |
| Campo Fernandez, B | 1 |
| Davila, A | 2 |
| Kohn, DB | 4 |
| Chang, VY | 1 |
| Cowan, MJ | 2 |
| Yu, J | 1 |
| Facchino, J | 2 |
| Fraser-Browne, C | 1 |
| Sanford, U | 1 |
| Kawahara, M | 1 |
| Dara, J | 2 |
| Long-Boyle, J | 2 |
| Oh, J | 1 |
| Chan, W | 1 |
| Chag, S | 1 |
| Broderick, L | 1 |
| Chellapandian, D | 1 |
| Decaluwe, H | 1 |
| Golski, C | 1 |
| Hu, D | 1 |
| Kuo, CY | 1 |
| Miller, HK | 1 |
| Petrovic, A | 1 |
| Currier, R | 1 |
| Hilton, JF | 1 |
| Punwani, D | 1 |
| Dvorak, CC | 2 |
| Malech, HL | 1 |
| McIvor, RS | 1 |
| Puck, JM | 2 |
| Bradford, KL | 1 |
| Liu, S | 1 |
| Krajinovic, M | 1 |
| Ansari, M | 1 |
| Garabedian, E | 1 |
| Tse, J | 1 |
| Wang, X | 1 |
| Shaw, KL | 1 |
| Gaspar, HB | 1 |
| Candotti, F | 2 |
| Cooper, AR | 1 |
| Lill, GR | 1 |
| Shaw, K | 1 |
| Carbonaro-Sarracino, DA | 1 |
| Sokolic, R | 1 |
| Pellegrini, M | 1 |
| Melton, A | 1 |
| Shimano, KA | 1 |
| Huang, JN | 1 |
| Dorsey, MJ | 1 |
| Chang, CK | 1 |
| Haddad, E | 2 |
| Leroy, S | 1 |
| Buckley, RH | 1 |
| Chevaleyre, J | 1 |
| Duchez, P | 1 |
| Rodriguez, L | 1 |
| Vlaski, M | 1 |
| Villacreces, A | 1 |
| Conrad-Lapostolle, V | 1 |
| Praloran, V | 1 |
| Ivanovic, Z | 1 |
| Brunet de la Grange, P | 1 |
| Cossellu, G | 1 |
| Seramondi, R | 1 |
| Benedicenti, S | 1 |
| Farronato, G | 1 |
| Olivi, G | 1 |
| Angiero, F | 1 |
| Cuellar-Rodriguez, J | 1 |
| Freeman, AF | 1 |
| Grossman, J | 1 |
| Su, H | 1 |
| Parta, M | 1 |
| Murdock, H | 1 |
| Shah, N | 1 |
| Bollard, C | 1 |
| Kong, HH | 1 |
| Moutsopoulos, N | 1 |
| Stone, K | 1 |
| Gea-Banacloche, J | 1 |
| Holland, SM | 1 |
| Hickstein, DD | 1 |
| Ünal, Ş | 1 |
| Tezcan, İ | 1 |
| Güçer, Ş | 1 |
| Boyraz, MS | 1 |
| Çağdaş, D | 1 |
| Uçkan Çetinkaya, D | 1 |
| Lin, M | 1 |
| Epport, K | 1 |
| Azen, C | 1 |
| Parkman, R | 1 |
| Shah, AJ | 1 |
| Kanegane, H | 1 |
| Taneichi, H | 1 |
| Nomura, K | 1 |
| Wada, T | 1 |
| Yachie, A | 1 |
| Imai, K | 1 |
| Ariga, T | 2 |
| Santisteban, I | 1 |
| Hershfield, MS | 1 |
| Miyawaki, T | 1 |
| Grunebaum, E | 1 |
| Daneman, A | 1 |
| Murguia-Favela, L | 1 |
| Manson, D | 1 |
| Kim, VH | 1 |
| Roifman, CM | 1 |
| Grunebaum, M | 1 |
| Fagioli, F | 1 |
| Biasin, E | 1 |
| Berger, M | 1 |
| Nesi, F | 1 |
| Saroglia, EH | 1 |
| Miniero, R | 1 |
| Martino, S | 1 |
| Tovo, PA | 1 |
| BOOTSMA, BK | 1 |
| LOPESCARDOZO, E | 1 |
| MOESCHLIN, S | 1 |
| Goebel, WS | 1 |
| Nelson, RP | 1 |
| Brahmi, Z | 1 |
| Gowan, DJ | 1 |
| Towell, PJ | 1 |
| Robertson, KA | 1 |
| Haut, PR | 1 |
| Gomez, L | 1 |
| Le Deist, F | 2 |
| Blanche, S | 2 |
| Cavazzana-Calvo, M | 2 |
| Griscelli, C | 1 |
| Fischer, A | 2 |
| Aucouturier, P | 1 |
| De Saint Basile, G | 1 |
| Bleyzac, N | 1 |
| Souillet, G | 1 |
| Magron, P | 1 |
| Janoly, A | 1 |
| Martin, P | 1 |
| Bertrand, Y | 1 |
| Galambrun, C | 1 |
| Dai, Q | 1 |
| Maire, P | 1 |
| Jelliffe, RW | 1 |
| Aulagner, G | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan[NCT00794508] | Phase 2 | 10 participants (Actual) | Interventional | 2008-11-30 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
As measured by ADA enzyme activity in peripheral blood mononuclear cells (NCT00794508)
Timeframe: 2 years
| Intervention | participants (Number) |
|---|---|
| Gamma-retroviral Mediated ADA Gene Transfer | 9 |
"Examine the safety of the procedure: harvesting bone marrow, isolating CD34+ hematopoietic stem/progenitor cells, performing ex vivo gene transduction with the MND-ADA gamma-retroviral vector, giving 90 mg/m2 busulfan to make space in the bone marrow to aid engraftment, and re-infusing the autologous gene-modified cells." (NCT00794508)
Timeframe: 2 years
| Intervention | participants (Number) | ||||
|---|---|---|---|---|---|
| Expected Grade 3 or 4 AE related to procedure | Unexpected Grade 3 or 4 AE related to procedure | Grade 3 or 4 AE related to study product | Alive | Deaths | |
| Gamma-retroviral Mediated ADA Gene Transfer | 10 | 9 | 0 | 10 | 0 |
As measured by quantitative polymerase chain reaction in peripheral blood cells separated into mononuclear and granulocyte fractions. (NCT00794508)
Timeframe: 2 years
| Intervention | participants (Number) | |
|---|---|---|
| For peripheral blood mononuclear cells | for peripheral blood granulocytes | |
| Gamma-retroviral Mediated ADA Gene Transfer | 10 | 4 |
2 reviews available for busulfan and Bare Lymphocyte Syndrome
| Article | Year |
|---|---|
B-cell reconstitution for SCID: should a conditioning regimen be used in SCID treatment?
Topics: Adenosine Deaminase; B-Lymphocytes; Bone Marrow Transplantation; Busulfan; CD3 Complex; Graft Surviv | 2013 |
[Gene therapy for primary immunodeficiency diseases].
Topics: Adaptor Proteins, Signal Transducing; Adenosine Deaminase; Busulfan; DNA-Binding Proteins; Genetic T | 2005 |
5 trials available for busulfan and Bare Lymphocyte Syndrome
| Article | Year |
|---|---|
Evaluation of clonal hematopoiesis in pediatric ADA-SCID gene therapy participants.
Topics: Busulfan; Child; Clonal Hematopoiesis; Genetic Therapy; Humans; Severe Combined Immunodeficiency | 2022 |
Lentiviral Gene Therapy for Artemis-Deficient SCID.
Topics: Antigens, CD34; B-Lymphocytes; Busulfan; DNA Repair Enzymes; Genetic Therapy; Genetic Vectors; Human | 2022 |
Cytoreductive conditioning intensity predicts clonal diversity in ADA-SCID retroviral gene therapy patients.
Topics: Adaptor Proteins, Signal Transducing; Adenosine Deaminase; Agammaglobulinemia; Antineoplastic Agents | 2017 |
Low Exposure Busulfan Conditioning to Achieve Sufficient Multilineage Chimerism in Patients with Severe Combined Immunodeficiency.
Topics: B-Lymphocytes; Busulfan; Child; Female; Follow-Up Studies; Humans; Infant; Male; Retrospective Studi | 2019 |
Matched related and unrelated donor hematopoietic stem cell transplantation for DOCK8 deficiency.
Topics: Adolescent; Adult; Busulfan; Child; Female; Graft Survival; Graft vs Host Disease; Guanine Nucleotid | 2015 |
15 other studies available for busulfan and Bare Lymphocyte Syndrome
| Article | Year |
|---|---|
Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.
Topics: Adenosine Deaminase; Agammaglobulinemia; Busulfan; Child; Genetic Therapy; Hematopoietic Stem Cell T | 2020 |
Busulfan administration flexibility increases the applicability of scid repopulating cell assay in NSG mouse model.
Topics: Animals; Body Weight; Busulfan; Cell Differentiation; Female; Graft Survival; Hematopoietic Stem Cel | 2013 |
Tooth developmental anomalies in severe combined immunodeficiency disease and juvenile myelomonocytic leukemia: common clinical features and treatment outcomes.
Topics: Adolescent; Antineoplastic Agents, Alkylating; Bone Marrow Transplantation; Busulfan; Child; Cycloph | 2013 |
Diagnosis: Melanoderma after Hematopoietic Stem Cell Transplantation.
Topics: Antilymphocyte Serum; Busulfan; Deferoxamine; Diagnosis, Differential; Ecchymosis; Humans; Infant; I | 2015 |
Long-term neurocognitive function of pediatric patients with severe combined immune deficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT).
Topics: Adaptation, Psychological; Alemtuzumab; Antibodies, Monoclonal; Antibodies, Monoclonal, Humanized; A | 2009 |
Successful bone marrow transplantation with reduced intensity conditioning in a patient with delayed-onset adenosine deaminase deficiency.
Topics: Adenosine Deaminase; Agammaglobulinemia; Bone Marrow Transplantation; Busulfan; Child, Preschool; Hu | 2013 |
Multiple osteochondromas following irradiation-containing conditioning in severe combined immunodeficiency.
Topics: Age Factors; Bone Marrow Transplantation; Bone Neoplasms; Busulfan; Cyclophosphamide; Humans; Infant | 2013 |
Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome.
Topics: Antilymphocyte Serum; Busulfan; Cyclosporine; Fetal Blood; Gastroenteritis; Graft vs Host Disease; H | 2003 |
[EXPERIENCE IN THE TREATMENT OF MALIGNANT DISEASES OF THE RETICULOENDOTHELIAL SYSTEM AND LEUKEMIAS. II].
Topics: Betamethasone; Busulfan; Chlorambucil; Copper; Cyclophosphamide; Dexamethasone; Genetic Diseases, X- | 1964 |
[CYTOSTATIC THERAPY].
Topics: Antineoplastic Agents; Breast Neoplasms; Busulfan; Chlorambucil; Colchicine; Cyclophosphamide; Femal | 1964 |
[MODERN TREATMENT OF LEUKEMIA].
Topics: Agranulocytosis; Busulfan; Chlorambucil; Cyclophosphamide; Drug Therapy; Genetic Diseases, X-Linked; | 1965 |
Serial transplantation resulting in tolerance to an unrelated cord blood graft.
Topics: Antilymphocyte Serum; B-Lymphocytes; Bone Marrow Transplantation; Busulfan; Chimerism; Cord Blood St | 2006 |
Treatment of Omenn syndrome by bone marrow transplantation.
Topics: Bone Marrow Transplantation; Busulfan; Child; Child, Preschool; Cyclophosphamide; Female; Graft Reje | 1995 |
Long-term chimerism and B-cell function after bone marrow transplantation in patients with severe combined immunodeficiency with B cells: A single-center study of 22 patients.
Topics: Antibody Formation; B-Lymphocytes; Bone Marrow Transplantation; Busulfan; Chimera; Cyclophosphamide; | 1999 |
Improved clinical outcome of paediatric bone marrow recipients using a test dose and Bayesian pharmacokinetic individualization of busulfan dosage regimens.
Topics: Adolescent; Alkylating Agents; Area Under Curve; Bayes Theorem; Bone Marrow Transplantation; Busulfa | 2001 |