Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome)
Conditions
Interventions
Treosulfan 14 g/m2, intravenous, day -6 -5 -4
Sponsors
Tokai University School of Medicine
1. School of Human Health Science Faculty of Medicine Kyoto University 2. Department of Pediatrics, Japanese Red cross Nagoya Daiichi Hospital 3. Department of Pediatrics, Nihon University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Mucopolysaccharidosis type I (Herler syndrome), type II (Hunter syndrome) 2. Body weight; equal or more than 10 kg 3. ECOG performance status; 0 or 1 4. The transplant should be the first one for the patient. Retransplantation is acceptable under the following conditions; after over 6 months from previous transplantation, and has no effect of previous preconditioning regimen 5. One of the following stem cell donor is available 1) 6/6 or 5/6 (either class I or class II) HLA-A/B/DR serologically matched related bone marrow 2) 6/6 HLA-A/B/DR allelic matched or HLA-DR/DRB1 serologically/allelic mismatched unrelated bone marrow 3) Cord blood units with 6/6, 5/6, or 4/6 HLA serologically match and nucleated cell dose equal or more than 3.5 x 10e7/kg, and CD34+ cell dose equal or more than 1.0 x 10e5/kg 6. Major organ dysfunction (laboratory data) 1) Ejection fraction at rest (UCG); equal or more than 50% 2) Arterial oxygen saturation without oxygen supplementation; equal or greater than 93% 3) Serum creatinine < 1.3 mg/dl 4) Total bilirubin <1.6 mg/dl or AST(GOT) < 2 x normal of each institution 7. Patients without following active infections 1) Pathogen-proven bacterial infection requiring antimicrobial treatment 2) Imaging study (XP, CT, US, MRI) manifested infectious foci requiring antimicrobial treatment 3) Abscess formation or necrotizing infection 4) Viral infection necessitating systemic antiviral agents 5) Culture-positive or PCR-positive tuberculosis/non-tuberculous mycobacterial infection 6) Pneumocystis pneumonia 7) Meningitis, Encephalitis, Encephalopathy 8) Protozoan infection 9) Intraocular fungal infection 8. No previous history of hypersensitivity to the following drugs that are used for conditioning or prophylaxis of GVHD Treosulfan (L-threitol-1,4-bis-methanesulfonate; dihydroxybusulfan) Fludarabine Antithymocyte globulin Cyclosporine Methotrexate Tacrolimus
Exclusion criteria
Exclusion criteria: 1. Down syndrome 2. HIV-positivity
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence of severe RRT (Grade III/IV) on day 28 after HSCT with Treosulfan as a conditioning regimen. | — |
Secondary
| Measure | Time frame |
|---|---|
| Regimen-related toxicity, engraftment rate, survival rate at 28 days posttransplant, survival rate at 100 days posttransplant, chimeric study, GVHD, hepatic SOS, event-free survival and overall survival at 1 year posttransplant. | — |
Countries
Japan
Contacts
Public ContactHiromasa Yabe
Tokai University School of Medicine Department of Cell Transplantation and Regenerative Medicine
Outcome results
None listed