Severe Combined Immunodeficiency, Stem Cell Transplant, Transplacental Maternal Engraftment
Conditions
Keywords
SCID, severe combined immunodeficiency, maternal engraftment, aGVHD prophylaxis, sirolimus
Brief summary
Study Design: SCID infants receiving an unconditioned haploidentical transplant will be started on Sirolimus (0.05 mg/kg/day) day -5 for Acute Graft-Versus-Host Disease (aGVHD) prophylaxis. Sirolimus levels will be monitored with goal sirolimus trough level of 5-8 ng/mL. Patients will be monitored for signs of aGVHD as defined by UCSF SOP CL 221.06 through day +100. Sirolimus will be tapered once T-regulatory cell to CD4 effector cell ratio is \> or = 9%. Setting: Inpatient BMT Unit Benioff Children's Hospital at UCSF Medical Center Study Subjects: 15 infants with diagnosis of maternally engrafted T cells SCID by CA Newborn screen receiving unconditioned haploidentical HSCT Main Outcome Measures: Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by SOP CL 221.06 by Day +100. Hypothesis 1. Patients placed on sirolimus prophylaxis will have lower incidence of aGVHD compared to historical controls. Hypothesis 2. Lower doses of sirolimus milligram per kilogram will be required to maintain goal troughs of 5-8 ng/mL.
Interventions
DRUGSirolimus
Sponsors
University of California, San Francisco
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Days to 1 Years
Healthy volunteers
No
Inclusion criteria
* Infants diagnosed with SCID on CA newborn screen * Evidence of Maternal Engraftment * Unconditioned haploidentical hematopoeitic stem cell transplant
Exclusion criteria
* Evidence of acute graft vs. host disease
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of aGVHD | 105 days | Incidence of aGVHD (dermatitis, hepatitis, enteritis) as defined by UCSF SOP CL 221.06 by Day +100. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| T-regulatory cell enumeration | 105 days | T-regulatory cell enumeration studies by blood draw prior to prophylaxis and at day +30, day+60, day+100. |
| Sirolimus therapeutic drug monitoring | 105 days | Sirolimus therapeutic drug monitoring will be done via blood draws(after initial 4th dose; thereafter every 4th dose with adjustment; once stable it will be measured weekly). We will determine the average dose in milligrams per kilograms that would be needed to reach therapeutic trough levels of 5-8 ng/mL. |
Countries
United States
Outcome results
None listed