Page last updated: 2024-10-24

busulfan and HbS Disease

busulfan has been researched along with HbS Disease in 25 studies

Research Excerpts

ExcerptRelevanceReference
"Thirty-four patients had thalassemia major and 4 had SCA."5.43Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience. ( Ben-Arush, M; Elhasid, R; Khalil, A; Rowe, JM; Zaidman, I, 2016)
"Busulfan (Bu) is an integral part of conditioning regimens for patients with sickle cell anemia (SCA) undergoing transplantation."2.80New insights into the pharmacokinetics of intravenous busulfan in children with sickle cell anemia undergoing bone marrow transplantation. ( Alfieri, C; Andreani, M; De Angelis, G; Dinallo, V; Gallucci, C; Gaziev, J; Ialongo, C; Isgrò, A; Lucarelli, G; Marziali, M; Mozzi, AF; Nguyen, L; Paciaroni, K; Petain, A; Ribersani, M; Sodani, P; Testi, M, 2015)
"Seven of 21 patients with sickle cell anemia developed neurologic complications 5 to 243 days (median, 33 days) after allogeneic marrow transplantation."2.68Neurologic complications after allogeneic marrow transplantation for sickle cell anemia. ( Bernaudin, F; Bunin, N; Johnson, FL; Lenarsky, C; Ohene-Frempong, K; Powars, D; Souillet, G; Sullivan, KM; Vannier, JP; Walters, MC, 1995)
"Thiotepa has improved engraftment when added to this regimen, however the minimum effective dose (MED) of thiotepa to achieve engraftment while minimizing toxicities has not been well established."1.91Feasibility study of busulfan, fludarabine, and thiotepa conditioning regimen for allogeneic hematopoietic stemcell transplantationfor children and young adults with nonmalignant disorders. ( Cascio, E; Castillo, P; Farhadfar, N; Fort, J; Fraser, A; Horn, B; Lamba, J; Moore-Higgs, G; Slayton, W, 2023)
"Busulfan was considered the cause in the first reported case because the transgene was not present in the AML/MDS."1.62Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither. ( DeBaun, MR; Jones, RJ, 2021)
" Conditioning with treosulfan alone at nonmyeloablative dosing (3."1.48Bone Marrow Transplantation after Nonmyeloablative Treosulfan Conditioning Is Curative in a Murine Model of Sickle Cell Disease. ( Devadasan, D; Goldman, FD; Pawlik, KM; Sun, CW; Townes, TM; Westin, ER; Wu, LC, 2018)
"Thirty-four patients had thalassemia major and 4 had SCA."1.43Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience. ( Ben-Arush, M; Elhasid, R; Khalil, A; Rowe, JM; Zaidman, I, 2016)
"Busulfan (BU) has a narrow therapeutic window and the average concentration of BU at steady state (Css) is critical for successful engraftment in children receiving BU as part of the preparative regimen for allogeneic transplants."1.40Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant. ( Brown, V; Bruce, K; Calder, C; Domm, J; Evans, M; Frangoul, H; Ho, R; Kassim, A; Maheshwari, S; Manes, B; Yang, E; Yeh, RF, 2014)
" Our experience shows that targeting busulfan AUC above the range used in previous multicenter trials appears safe and may contribute to sustained engraftment in SCD."1.37Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease. ( Chiang, KY; Haight, AE; Horan, J; Hutcherson, D; McPherson, ME; Olson, E, 2011)

Research

Studies (25)

TimeframeStudies, this research(%)All Research%
pre-19900 (0.00)18.7374
1990's5 (20.00)18.2507
2000's2 (8.00)29.6817
2010's15 (60.00)24.3611
2020's3 (12.00)2.80

Authors

AuthorsStudies
Fraser, A1
Castillo, P1
Cascio, E1
Moore-Higgs, G1
Farhadfar, N1
Fort, J1
Slayton, W1
Lamba, J1
Horn, B1
Remy, A1
Théorêt, Y1
Ansari, M1
Bittencourt, H1
Ducruet, T1
Nava, T1
Pastore, Y1
Rezgui, MA1
Krajinovic, M1
Kleiber, N1
Jones, RJ1
DeBaun, MR1
Pawlowska, AB1
Cheng, JC1
Karras, NA1
Sun, W1
Wang, LD1
Bell, AD1
Gutierrez, L1
Rosenthal, J1
Rangarajan, HG1
Abu-Arja, R1
Pai, V1
Guilcher, GMT1
Soni, S2
Devadasan, D1
Sun, CW1
Westin, ER1
Wu, LC1
Pawlik, KM1
Townes, TM1
Goldman, FD1
Demirci, S1
Mora, JJH1
Yapundich, M1
Drysdale, C1
Gamer, J1
Nassehi, T1
Bonifacino, AC1
Krouse, AE1
Linde, NS1
Donahue, RE1
Tisdale, JF1
Uchida, N1
Radhakrishnan, K2
Bhatia, M2
Geyer, MB2
Del Toro, G2
Jin, Z2
Baker, C2
Harrison, L1
Morris, E2
Baxter-Lowe, LA2
Cairo, MS2
Maheshwari, S1
Kassim, A1
Yeh, RF1
Domm, J1
Calder, C1
Evans, M1
Manes, B1
Bruce, K1
Brown, V1
Ho, R1
Frangoul, H1
Yang, E1
Gross, TG1
Rangarajan, H1
Baker, KS1
Sturm, M1
Rhodes, M1
Satwani, P1
George, D1
Garvin, J1
Zuckerman, W1
Lee, MT1
Licursi, M1
Hawks, R1
Smilow, E1
Schwartz, J1
Gaziev, J1
Isgrò, A1
Mozzi, AF1
Petain, A1
Nguyen, L1
Ialongo, C1
Dinallo, V1
Sodani, P1
Marziali, M1
Andreani, M1
Testi, M1
Paciaroni, K1
Gallucci, C1
De Angelis, G1
Alfieri, C1
Ribersani, M1
Lucarelli, G1
Horan, JT1
Haight, A1
Dioguardi, JL1
Brown, C1
Grizzle, A1
Shelman, C1
Kanter, J1
Hale, G1
Nieder, M1
Benton, M1
Kasow, KA1
Abraham, A1
Chiang, KY2
Strocchio, L1
Zecca, M1
Comoli, P1
Mina, T1
Giorgiani, G1
Giraldi, E1
Vinti, L1
Merli, P1
Regazzi, M1
Locatelli, F1
Elchuri, SV1
Williamson, RS1
Clark Brown, R1
Haight, AE2
Spencer, JB1
Buchanan, I1
Hassen-Schilling, L1
Brown, MR1
Mertens, AC1
Meacham, LR1
Zaidman, I1
Rowe, JM1
Khalil, A1
Ben-Arush, M1
Elhasid, R1
McPherson, ME1
Hutcherson, D1
Olson, E1
Horan, J1
Donnez, J1
Squifflet, J1
Pirard, C1
Jadoul, P1
Dolmans, MM1
Kean, LS2
Manci, EA1
Perry, J1
Balkan, C1
Coley, S1
Holtzclaw, D1
Adams, AB2
Larsen, CP2
Hsu, LL2
Archer, DR2
Walters, MC1
Sullivan, KM1
Bernaudin, F3
Souillet, G2
Vannier, JP2
Johnson, FL1
Lenarsky, C1
Powars, D1
Bunin, N1
Ohene-Frempong, K1
Vergauwen, P1
Ferster, A1
Valsamis, J1
Chanoine, JP1
Plouvier, E1
Lemerle, S1
Michel, G1
Bordigoni, P1
Lutz, P1
Kuentz, M2
Hernigou, P1
Reinert, P1
Vernant, JP1
Durham, MM1
Perry, JR1
Dillehay, D1
Pearson, TC1
Waller, EK1
Vermylen, C1
Cornu, G1
Philippe, M1
Ninane, J1
Borja, A1
Latinne, D1
Ferrant, A1
Michaux, JL1
Sokal, G1

Clinical Trials (2)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
A Phase 1/2 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Severe Sickle Cell Disease[NCT02140554]Phase 1/Phase 250 participants (Anticipated)Interventional2014-08-31Active, not recruiting
Allogeneic Stem Cell Transplant to Induce Mixed Donor Chimerism in Patients With Sickle Cell Disease and Thalassemia[NCT00408447]Phase 253 participants (Actual)Interventional2004-09-30Active, not recruiting
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trials

4 trials available for busulfan and HbS Disease

ArticleYear
Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease.
    Bone marrow transplantation, 2014, Volume: 49, Issue:7

    Topics: Adolescent; Adult; Alemtuzumab; Anemia, Sickle Cell; Antibodies, Monoclonal, Humanized; Antineoplast

2014
New insights into the pharmacokinetics of intravenous busulfan in children with sickle cell anemia undergoing bone marrow transplantation.
    Pediatric blood & cancer, 2015, Volume: 62, Issue:4

    Topics: Adolescent; Allografts; Anemia, Sickle Cell; Bone Marrow Transplantation; Busulfan; Child; Child, Pr

2015
Using fludarabine to reduce exposure to alkylating agents in children with sickle cell disease receiving busulfan, cyclophosphamide, and antithymocyte globulin transplant conditioning: results of a dose de-escalation trial.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2015, Volume: 21, Issue:5

    Topics: Adolescent; Allografts; Anemia, Sickle Cell; Antilymphocyte Serum; Busulfan; Child; Child, Preschool

2015
Neurologic complications after allogeneic marrow transplantation for sickle cell anemia.
    Blood, 1995, Feb-15, Volume: 85, Issue:4

    Topics: Adolescent; Adult; Anemia, Sickle Cell; Anticonvulsants; Antilymphocyte Serum; Bone Marrow Transplan

1995

Other Studies

21 other studies available for busulfan and HbS Disease

ArticleYear
Feasibility study of busulfan, fludarabine, and thiotepa conditioning regimen for allogeneic hematopoietic stemcell transplantationfor children and young adults with nonmalignant disorders.
    Pediatric blood & cancer, 2023, Volume: 70, Issue:7

    Topics: Anemia, Sickle Cell; Busulfan; Child; Feasibility Studies; Hematopoietic Stem Cell Transplantation;

2023
Is Busulfan Clearance Different in Patients With Sickle Cell Disease? Let's Clear Up That Case With Some Controls.
    Journal of pediatric hematology/oncology, 2021, 08-01, Volume: 43, Issue:6

    Topics: Adolescent; Adult; Anemia, Sickle Cell; Busulfan; Case-Control Studies; Child; Child, Preschool; Cro

2021
Leukemia after gene therapy for sickle cell disease: insertional mutagenesis, busulfan, both, or neither.
    Blood, 2021, 09-16, Volume: 138, Issue:11

    Topics: Alkylating Agents; Anemia, Sickle Cell; Busulfan; Clinical Trials as Topic; Gene Editing; Genetic Th

2021
HLA Haploidentical Stem Cell Transplant with Pretransplant Immunosuppression for Patients with Sickle Cell Disease.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2018, Volume: 24, Issue:1

    Topics: Adolescent; Anemia, Sickle Cell; Antilymphocyte Serum; Busulfan; Cohort Studies; Dexamethasone; Graf

2018
Outcomes of Unrelated Donor Stem Cell Transplantion with Post-Transplant Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients with Severe Sickle Cell Disease.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2018, Volume: 24, Issue:2

    Topics: Adolescent; Anemia, Sickle Cell; Antilymphocyte Serum; Antineoplastic Combined Chemotherapy Protocol

2018
Bone Marrow Transplantation after Nonmyeloablative Treosulfan Conditioning Is Curative in a Murine Model of Sickle Cell Disease.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2018, Volume: 24, Issue:8

    Topics: Anemia, Sickle Cell; Animals; Antibodies; Antineoplastic Agents, Alkylating; Bone Marrow Transplanta

2018
Fetal hemoglobin and F-cell variance in mobilized CD34
    Experimental hematology, 2019, Volume: 75

    Topics: Allografts; Anemia, Sickle Cell; Animals; Antigens, CD34; Busulfan; Erythrocytes, Abnormal; Fetal He

2019
Busulfan, fludarabine, and alemtuzumab conditioning and unrelated cord blood transplantation in children with sickle cell disease.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2013, Volume: 19, Issue:4

    Topics: Alemtuzumab; Anemia, Sickle Cell; Antibodies, Monoclonal, Humanized; Busulfan; Child; Child, Prescho

2013
Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant.
    Bone marrow transplantation, 2014, Volume: 49, Issue:3

    Topics: Adolescent; Age Factors; Anemia, Sickle Cell; Antilymphocyte Serum; Bone Marrow Transplantation; Bus

2014
Outcomes of matched sibling donor hematopoietic stem cell transplantation for severe sickle cell disease with myeloablative conditioning and intermediate-dose of rabbit anti-thymocyte globulin.
    Pediatric blood & cancer, 2014, Volume: 61, Issue:9

    Topics: Adolescent; Anemia, Sickle Cell; Animals; Antilymphocyte Serum; Busulfan; Child; Child, Preschool; F

2014
Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in children with sickle cell disease.
    British journal of haematology, 2015, Volume: 169, Issue:5

    Topics: Adolescent; Anemia, Sickle Cell; Antineoplastic Combined Chemotherapy Protocols; Busulfan; Child; Ch

2015
The effects of hydroxyurea and bone marrow transplant on Anti-Müllerian hormone (AMH) levels in females with sickle cell anemia.
    Blood cells, molecules & diseases, 2015, Volume: 55, Issue:1

    Topics: Adolescent; Anemia, Sickle Cell; Anti-Mullerian Hormone; Antisickling Agents; Biomarkers; Bone Marro

2015
Allogeneic Stem Cell Transplantation in Congenital Hemoglobinopathies Using a Tailored Busulfan-Based Conditioning Regimen: Single-Center Experience.
    Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2016, Volume: 22, Issue:6

    Topics: Adolescent; Adult; Anemia, Sickle Cell; beta-Thalassemia; Busulfan; Child; Child, Preschool; Chimeri

2016
Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease.
    Bone marrow transplantation, 2011, Volume: 46, Issue:1

    Topics: Adolescent; Anemia, Sickle Cell; Bone Marrow Transplantation; Busulfan; Child; Child, Preschool; Chi

2011
Restoration of ovarian function after allografting of ovarian cortex between genetically non-identical sisters.
    Human reproduction (Oxford, England), 2010, Volume: 25, Issue:10

    Topics: Adolescent; Adult; Anemia, Sickle Cell; Antineoplastic Agents, Alkylating; beta-Thalassemia; Busulfa

2010
Chimerism and cure: hematologic and pathologic correction of murine sickle cell disease.
    Blood, 2003, Dec-15, Volume: 102, Issue:13

    Topics: Anemia, Sickle Cell; Animals; Bone Marrow; Bone Marrow Transplantation; Busulfan; Cell Survival; Dis

2003
Primary ovarian failure after prepubertal marrow transplant in a girl.
    Lancet (London, England), 1994, Jan-08, Volume: 343, Issue:8889

    Topics: Adolescent; Anemia, Sickle Cell; Bone Marrow Transplantation; Busulfan; Cyclophosphamide; Female; Hu

1994
Bone marrow transplantation (BMT) in 14 children with severe sickle cell disease (SCD): the French experience. GEGMO.
    Bone marrow transplantation, 1993, Volume: 12 Suppl 1

    Topics: Adolescent; Africa; Anemia, Sickle Cell; Bone Marrow Purging; Bone Marrow Transplantation; Busulfan;

1993
Bone-marrow transplantation in sickle-cell disease. Effect on osteonecrosis: a case report with a four-year follow-up.
    The Journal of bone and joint surgery. American volume, 1997, Volume: 79, Issue:11

    Topics: Adolescent; Anemia, Hemolytic; Anemia, Sickle Cell; Bone Marrow Transplantation; Bone Regeneration;

1997
A cure for murine sickle cell disease through stable mixed chimerism and tolerance induction after nonmyeloablative conditioning and major histocompatibility complex-mismatched bone marrow transplantation.
    Blood, 2002, Mar-01, Volume: 99, Issue:5

    Topics: Abatacept; Anemia, Sickle Cell; Animals; Antibodies; Antigens, CD; Antigens, Differentiation; Bone M

2002
Bone marrow transplantation in sickle cell anaemia.
    Archives of disease in childhood, 1991, Volume: 66, Issue:10

    Topics: Adolescent; Adult; Anemia, Sickle Cell; Bone Marrow Transplantation; Busulfan; Child; Child, Prescho

1991