busulfan has been researched along with Mucopolysaccharidosis I in 9 studies
Mucopolysaccharidosis I: Systemic lysosomal storage disease caused by a deficiency of alpha-L-iduronidase (IDURONIDASE) and characterized by progressive physical deterioration with urinary excretion of DERMATAN SULFATE and HEPARAN SULFATE. There are three recognized phenotypes representing a spectrum of clinical severity from severe to mild: Hurler syndrome, Hurler-Scheie syndrome and Scheie syndrome (formerly mucopolysaccharidosis V). Symptoms may include DWARFISM; hepatosplenomegaly; thick, coarse facial features with low nasal bridge; corneal clouding; cardiac complications; and noisy breathing.
| Excerpt | Relevance | Reference |
|---|---|---|
| " Median (range) oral clearance corrected for bioavailability (Cl/F in ml/min/kg), area under the curve (AUC in ng min/ml) and BU plasma concentration (Cp in ng/ml) with the fourth dose were 5." | 2.70 | Oral busulfan pharmacokinetics and engraftment in children with Hurler syndrome and other inherited metabolic storage diseases undergoing hematopoietic cell transplantation. ( Abel, S; DeFor, TE; Jacobson, P; Krivit, W; Park, JJ; Peters, C; Thrall, M, 2001) |
| "Busulfan is an alkylating agent routinely used in conditioning regimens prior to allogeneic hematopoietic cell transplantation (HCT) for various nonmalignant disorders, including inborn errors of metabolism." | 1.56 | Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients. ( Dvorak, CC; Kharbanda, S; Long-Boyle, J; Shukla, P, 2020) |
| "For patients with mucopolysaccharidosis type IH (MPS1-H; Hurler syndrome), early allogeneic hematopoietic stem cell transplantation (HSCT) is the treatment of choice." | 1.40 | Unrelated CD3/CD19-depleted peripheral stem cell transplantation for Hurler syndrome. ( Benesch, M; Brunner-Krainz, M; Lackner, H; Paschke, E; Plecko, B; Raicht, A; Schwinger, W; Seidel, M; Sovinz, P; Sperl, D; Strenger, V; Urban, C, 2014) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 4 (44.44) | 29.6817 |
| 2010's | 3 (33.33) | 24.3611 |
| 2020's | 2 (22.22) | 2.80 |
| Authors | Studies |
|---|---|
| Shukla, P | 1 |
| Dvorak, CC | 1 |
| Long-Boyle, J | 1 |
| Kharbanda, S | 1 |
| Lum, SH | 1 |
| Orchard, PJ | 2 |
| Lund, TC | 2 |
| Miller, WP | 2 |
| Boelens, JJ | 3 |
| Wynn, R | 3 |
| Schwinger, W | 1 |
| Sovinz, P | 1 |
| Benesch, M | 1 |
| Lackner, H | 1 |
| Seidel, M | 1 |
| Strenger, V | 1 |
| Sperl, D | 1 |
| Raicht, A | 1 |
| Brunner-Krainz, M | 1 |
| Paschke, E | 1 |
| Plecko, B | 1 |
| Urban, C | 1 |
| Mallhi, K | 1 |
| Cao, Q | 1 |
| Tolar, J | 1 |
| Rocha, V | 1 |
| Aldenhoven, M | 1 |
| O'Meara, A | 2 |
| Michel, G | 1 |
| Ionescu, I | 1 |
| Parikh, S | 1 |
| Prasad, VK | 1 |
| Szabolcs, P | 1 |
| Escolar, M | 1 |
| Gluckman, E | 1 |
| Cavazzana-Calvo, M | 2 |
| Kurtzberg, J | 1 |
| Gardner, CJ | 1 |
| Robinson, N | 1 |
| Meadows, T | 1 |
| Will, A | 1 |
| Mercer, J | 1 |
| Church, HJ | 1 |
| Tylee, K | 1 |
| Wraith, JE | 2 |
| Clayton, PE | 1 |
| Grigull, L | 1 |
| Beilken, A | 1 |
| Schrappe, M | 1 |
| Das, A | 1 |
| Luecke, T | 1 |
| Sander, A | 1 |
| Stanulla, M | 1 |
| Rehe, K | 1 |
| Sauer, M | 1 |
| Schmid, H | 1 |
| Welte, K | 1 |
| Lukacs, Z | 1 |
| Gal, A | 1 |
| Sykora, KW | 2 |
| Wynn, RF | 1 |
| Veys, P | 1 |
| Bertrand, Y | 1 |
| Souillet, G | 1 |
| Fischer, A | 1 |
| Sedlacek, P | 1 |
| Rovelli, A | 1 |
| Uiterwaal, CS | 1 |
| Wulffraat, N | 1 |
| Jacobson, P | 1 |
| Park, JJ | 1 |
| DeFor, TE | 1 |
| Thrall, M | 1 |
| Abel, S | 1 |
| Krivit, W | 1 |
| Peters, C | 1 |
2 trials available for busulfan and Mucopolysaccharidosis I
| Article | Year |
|---|---|
Transplantation of allogeneic CD34-selected stem cells after fludarabine-based conditioning regimen for children with mucopolysaccharidosis 1H (M. Hurler).
Topics: Antigens, CD34; Antilymphocyte Serum; Busulfan; Child, Preschool; Graft Survival; Hematopoiesis; Hem | 2005 |
Oral busulfan pharmacokinetics and engraftment in children with Hurler syndrome and other inherited metabolic storage diseases undergoing hematopoietic cell transplantation.
Topics: Administration, Oral; Adolescent; Adult; Area Under Curve; Busulfan; Child; Child, Preschool; Combin | 2001 |
7 other studies available for busulfan and Mucopolysaccharidosis I
| Article | Year |
|---|---|
Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients.
Topics: Area Under Curve; Busulfan; Child; Chimerism; Female; Hematopoietic Stem Cell Transplantation; Hemat | 2020 |
Outcome After Cord Blood Transplantation Using Busulfan Pharmacokinetics-Targeted Myeloablative Conditioning for Hurler Syndrome.
Topics: Busulfan; Child; Cord Blood Stem Cell Transplantation; Humans; Mucopolysaccharidosis I; Retrospectiv | 2021 |
Unrelated CD3/CD19-depleted peripheral stem cell transplantation for Hurler syndrome.
Topics: Antigens, CD19; Antineoplastic Agents, Alkylating; Busulfan; CD3 Complex; Chimerism; Drug-Related Si | 2014 |
Non-myeloablative conditioning for second hematopoietic cell transplantation for graft failure in patients with non-malignant disorders: a prospective study and review of the literature.
Topics: Adolescent; Adrenoleukodystrophy; Busulfan; Child; Child, Preschool; Graft Rejection; Graft Survival | 2017 |
Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome.
Topics: Age Factors; Antilymphocyte Serum; Busulfan; Cell Count; Cord Blood Stem Cell Transplantation; Cyclo | 2009 |
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H).
Topics: Adolescent; Adult; Body Height; Busulfan; Child; Cyclophosphamide; Endocrine System; Endocrine Syste | 2011 |
Outcomes of hematopoietic stem cell transplantation for Hurler's syndrome in Europe: a risk factor analysis for graft failure.
Topics: Busulfan; Child; Child, Preschool; Databases, Factual; Disease-Free Survival; Europe; Female; Graft | 2007 |