Table 2. Patients Characteristics and Response to VST Therapy.
| Center | P # | Dx | Age at HSCT |
Donor | Time from HSCT to VST (mo) |
Viral infections prior to VST |
Viral status at time of VST infusion |
Prior antiviral tmts |
VST source and specificity |
Immuno- suppress- ion at time of VST therapy |
VST dose |
Antiviral response |
Adverse Events |
Follow- up time (years post- HSCT) |
Current status |
Study # / Ref |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| BCM | 1 | IL7RA-SCID | 6 mo | 6/6 UCB | 4.5 | None | N/A | None | Cord blood, CMV/EBV/ADV | CSA, prednisone | 1 dose of 1.5×10E7/m2 | No detectable EBV, ADV, CMV | None | 2 yrs 7 mo | Alive & well | NCT008 80789 / Unpubl. |
| 2 | IL2RG-SCID | 8 mo | 5/6 UCB | 4.5 | None | N/A | None | Cord blood, CMV/EBV/ADV | CSA/prednisone | 1 dose of 2.5×10E7/m2 | No detectable EBV, ADV CMV | None | 2 yrs 4 mo | Alive & well | NCT008 80789 / Unpubl. | |
| 3 | IL2RG-SCID | 2 mo | 5/6 UCB | 3 | None | N/A | None | Cord blood, CMV/EBV/ADV | MMF/prednisone | 1 dose of 1×10E7/m2 | No detectable EBV, ADV CMV | None | 3 yrs 2 mo | Alive & well | NCT008 80789 / Unpubl. | |
| 4 | IL2RG-SCID | 2 mo | MMRD (Maternal haploidentical) | 9 | None | N/A | None | HSCT Donor, CMV/EBV/ADV | None | 1 dose of 1×10E7/m2 | No detectable EBV, ADV CMV | None | 4 yr 4 mo | Alive & well | NCT000 78533 / Ref 20 | |
| 5 | SCID, unknown gene | 1 yr | 10/10 MUD | 2 | None | N/A | None | HSCT Donor, ADV | CSA, prednisone | 1 dose of 1.35×10E8/m2 | No detectable ADV | None | 11 mo | Alive & well | NCT005 90083 / Ref 21 | |
| 6 | MHC II deficiency | 10 mo | 10/10 MRD | 1.5 | CMV | CMV viremia, pneumonitis | Foscarnet, Ganciclovir, Cytogam | HSCT Donor, CMV/EBV/ADV | CSA | 1 dose of 1×10E7/m2 | CMV - CR | None | 2 yrs 10 mo | Alive & well | NCT000 78533 / Ref 20 | |
| 7 | WAS | 8 mo | 9/10 MMUD | 5 | None | N/A | None | HSCT Donor, CMV/EBV/ADV | None | 1 dose of 1×10e7/m2 | No detectable EBV, ADV CMV | None | 5 yrs 3 mo | Alive & well | NCT000 78533 / Ref 20 | |
| 8 | WAS | 1.2 yrs | 10/10 MUD | 2.3 | EBV | EBV viremia | Rituximab | Donor EBV | CSA | 1 dose of 2×10E7/m2 | EBV – CR | None | 3 yr 9 mo | Alive and well | NCT000 58812 / Ref 24 | |
| 9 | NK defect/ SCAEBV | 12 yrs | 7/10 MMUD | 7 | EBV | EBV viremia | Foscarnet for prior CMV | HSCT Donor, EBV | None | 1 dose of 1×10E8/m2 | EBV - CR | None | 4 yrs | Alive & well | NCT000 58812 / Ref 24 | |
| 10 | SCAEBV | 6.9 yrs | MRD (syngeneic) | 2.5 | EBV | EBV viremia | None | Donor EBV | None | 1 dose of 2×10E7/m2 | EBV – PR | None | 4 yrs 6 mo | Died -progressive T-cell lymphoma | NCT000 58812 / Ref 24 | |
| 11 | SCAEBV | 10.6 yrs | 10/10 MUD | 12 | EBV-LPD | EBV resolved | Rituximab | Donor EBV | None | 1 dose of 2×10E7/m2 | No further EBV reactivation | None | 10 yr 3 mo | Alive and well | NCT000 58812 / Ref 24 | |
| 12 | XLP (SLAM mutation) | 19 mo | 8/8 MUD | 3 | EBV | EBV viremia | None | HSCT Donor, EBV | CSA, prednisone | 1 dose of 2×10E7/m2 | EBV-CR | None | 9 yrs 5 mo | Alive & well | NCT000 58812 / Ref 24 | |
| 13 | XLP and lymphoma | 16 yrs | 9/10 MMUD | 2 | None | N/A | None | HSCT Donor, EBV | CSA | 1 dose of 2×10E7/m2 | No detectable EBV | None | 1 yr 6 mo | Alive & well | NCT000 58812 / Ref 24 | |
| 14 | LAD1 | 6 mo | 10/10 MRD | 2 | ADV | ADV viremia | Cidofovir | HSCT Donor, CMV/EBV/ADV/HHV6/BK | FK | 1 dose of 2×10E7/m2 | ADV - CR | None | 1 yr 7 mo | Alive & well | NCT015 70283 / Ref 23 | |
| 15 | GATA2 | 18 yrs | 9/10 MMUD | 3 | BK, CMV | BK viremia | Foscarnet, ValGanciclovir | HSCT Donor, CMV/EBV/ADV/HHV6/BK | FK/prednisone | 1 dose of 2×10E7/m2 | BK - CR; EBV - CR * | None | 1 yr 11mo | Alive & well | NCT015 70283 / Ref 23 | |
| 16 | HLH | 19 yrs | 10/10 -MUD | 2 | HHV6, BK | HHV6, BK viremia | None | HSCT Donor, CMV/EBV/ADV/HHV6/BK | MMF/prednisone | 1 dose of 1×10E7/m2 | HHV6 - CR; BK- NR, EBV-CR* | None | 1 yr 10 mo | Alive & well | NCT015 70283 / Ref 23 | |
| GOSH | 17 | CID/enterocolitis | 5.9 yrs | 10/10 MUD | 5.3 | EBV-LPD | EBV resolved | COP/Rituxima b | Donor EBV | Prednisolone | 1 dose of 2.5×10E7/m2 | No further EBV reactivation | None | 13 years 1 month | Alive & well | EIND / Ref 24 |
| 18 | CID/enterocolitis | 4.7 yrs | 10/10 MUD | 3.7 | None | N/A | None | Donor EBV | Methylpred | 1 dose of 2.5×10E7/m2 | No detectable EBV | None | 11 years 2 months | Alive & well | EIND / Ref 24 | |
| 19 | WAS | 17.7 yrs | 10/10 MUD | 4.3 | EBV-LPD | EBV resolved | Rituximab | Donor EBV | None | 1 dose of 2.5×10E7/m2 | No further EBV reactivation | None | 13 years 2 months | Alive & well | EIND / Ref 24 | |
| 20 | XLP | 12.4 yrs | 9/10 MMUD | 2.4 | EBV | EBV resolved | Rituximab | Donor EBV | CSA, MMF | 1 dose of 2.0×10E7/m2 | No further EBV reactivation | None | 10 years 6 months | Alive & well | EIND / Ref 24 | |
| 21 | XLP | 11.8 yrs | 9/10 MMUD | 3.3 | EBV | EBV viremia | None | Donor EBV | CSA | 1 dose of 2.0×10E7/m2 | EBV - PR | None | 11 years | Alive & well | EIND / Ref 24 | |
| 22 | XLP | 8.7 yrs | 9/10 MMUD | 2.8 | EBV | EBV resolved | Rituximab | Donor EBV | CSA | 1 dose of 2.0×10E7/m2 | EBV - CR* | None | 10 years 1 month | Alive & well | EIND / Ref 24 | |
| 23 | XLP | 11.8 yrs | 9/10 MMUD | 4 | EBV-LPD | EBV viremia | COP/COP AD | Donor EBV | CSA | 1 dose of 2.0×10E7/m2 | EBV - PR | None | 9 years 2 months | Alive & well | EIND / Ref 24 | |
| 24 | XLP-like | 5.4 yrs | 9/10 MMUD | 2.8 | EBV | EBV resolved | Rituximab | Donor EBV | CSA, Prednisolone | 1 dose of 2.0×10E7/m2 | No further EBV reactivation | None | 10 years 1 month | Alive & well | EIND / Ref 24 | |
| CNMC | 25 | ADA-SCID | N/A | N/A | Pre-transplant (6 yrs old) | EBV, CMV | EBV-LPD | Rituximab, ganciclovir | Third party (MMUD, 5/10 match), CMV/EBV/ADV | None | 1 dose of 5 × 10E6/m2 | EBV - NR | None | N/A | Died -EBV lymphoma, D+110 | NCT025 10404 / Unpubl. |
| 26 | SCID, unknown gene | 5 mo | MMRD (Maternal 7/10) | 1 | CMV | CMV viremia | Ganciclovir, Foscarnet, Cidofovir | HSCT Donor (7/10 match) CMV/EBV/ADV | MMF | 2 doses of 5×10E6/m2 | CMV - PR | None | 1 yr | Alive & well | NCT019 45814 / Unpubl. | |
| 27 | SCID, unknown gene | 4 mo | MMRD (Maternal 7/12) | 1 | CMV | CMV viremia | Foscarnet, Cidofovir, Cytogam | HSCT Donor, CMV/EBV/ADV | None | 1 dose of 1 × 10E7/m2 | CMV-NR | None | N/A | Died, refractory CMV and disseminated BCG, D+92 | NCT019 45814 / Unpubl. | |
| 2.5 | CMV viremia and pneumonitis | Third-party (MMUD, 4/12 match), CMV/EBV/ADV | None | 1 dose of 2 × 10E7/m2 | N/A | N/A | <2 weeks | |||||||||
| 28 | MHC II deficiency | 7 mo | MRD | 2 | None | N/A | Acyclovir (ppx) | HSCT Donor, CMV/EBV/ADV | CSA/prednisone | 1 dose of 5×10E6/m2 | CMV - CR* | aGVHD grade II, pericardial effusion | 10 mo | Alive & well | NCT019 45814 / Unpubl. | |
| 29 | HLH (STXBP2) | 4 yrs | 8/10 MMUD | 1 | CMV, EBV | CMV, EBV viremia | Ganciclovir, Rituximab | HSCT Donor, CMV/EBV/ADV | FK, prednisone | 2 doses of 1×10E7/m2 | CMV-CR, EBV-CR | Grade I cGVHD | 4 mo | Alive, grade I cGVHD | NCT019 45814 / Unpubl | |
| 30 | WAS | 17 months | 9/10 MMUD | 2 | None | N/A | None | HSCT Donor, CMV/EBV/ADV | FK/prednisone | 1 dose of 2 × 10E7/m2 | EBV-CR* | None | 2 months | Alive & well | NCT019 45814 / Unpubl. | |
| 31 | IL-10R Deficiency | 10 mo | MMRD (Maternal 6/8) | 0.7 | CMV, ADV, HBV | ADV viremia /pneumonia | Cidofovir, Foscarnet | Third-party (Paternal, 6/8 match), CMV/EBV/ADV | None | 1 dose of 2×10E7/m2 | ADV - CR, CMV-CR* | Acute GVHD, grade II gut | 5 mo | Alive, ongoing GVHD | EIND / Unpubl. | |
| NCL | 32 | CGD | 12 yrs | 11/12 MMUD | 27 | BK, CMV, EBV (urine) HHV6, ADV (blood), EBV-LPD | EBV-LPD | Acyclovir, rituximab | Third party (MMUD, 3/10 match), EBV | Methylpred | 4 doses of 2×10E6/kg (5.9×10E7/m2) | EBV - NR | None | 0.3 | Died -PTLD | EIND/Ref 25 |
| 33 | CTPS1 | 15 mo | 10/10 UCD | 1 month pre-HSCT | EBV (pre-transplant LPD), RSV, ADV | EBV-LPD | Acyclovir, rituximab | Third party (MMUD, 9/10 match), EBV | None, though received COP prior to VST* | 2 doses of 2×10E6/kg (3.9×10E7/m2) | EBV - CR | None | 2.5 yrs | Alive & well | EIND/Ref 25 | |
| 34 | HLH | 7 yrs | MUD | 1.7 | HHV6, EBV | EBV viremia | Foscarnet, Acyclovir, rituximab | Third party (MMUD, 5/10 match), EBV | CSA/MMF/Methylpred/infliximab | 3 doses of 2×10E6/kg (6.2×10E7/m2) | EBV - PR | Cerebral edema | Died 26 days after first VST dose | Died, EBV-PTLD with cerebral edema, D+76 | EIND/Unpubl. | |
| 35 | CID | 3.7 yrs | 9/10 MMUD (HLA-DQmm) | 5 | ADV HHV6, EBV-LPD | EBV-LPD | Acyclovir, cidofovir, rituximab, COP | Third party (MMUD, 3/10 match) EBV | Methylpred/FK/infliximab/ECP | 4 doses of 2×10E6/kg (4.6×10E7/m2) | EBV - NR | None | N/A | Died -PTLD | EIND/Unpubl. | |
| 36 | CD4 lymphopenia | 13 mo | MMRD (Maternal 5/10) | 8 days | Multi-drug resistant CMV | CMV-viremia | Ganciclovir, Foscarnet, Cidofovir | HSCT Donor, CMV-selected cells | CSA/MMF | 1 dose of 2×10E6/kg (3.8×10E7/m2) | CMV-PR | GVHD, grade III skin | D+280 | Died of fungal pneumonia, D+280 | EIND/Unpubl. |
Abbreviations: P#: Patient #; TMTS: treatments; BCM: Baylor College of Medicine; CNMC: Children's National Medical Center; NCL: Newcastle Hospital; GOSH: Great Ormond Street Hospital; UCB: Umbilical cord blood, MMRD: Mismatched related donor; MRD: matched related donor; MMUD: mismatched unrelated donor; MUD: matched unrelated donor; ADV: adenovirus; LPD: lymphoproliferative disease; COP: cyclophosphamide/ vincristine/ prednisone; COPAD: cyclophosphamide/vincristine/ prednisolone/doxorubicin; CSA: Cyclosporin A; MMF: Mycophenylate mofetil; FK: Tacrolimus; CR: complete response; PR: partial response; NR: no response; BCG: Bacillus-calmette-guerin; PTLD: post-transplantation lymphoproliferative disease; Mo: months; Yrs: years.
*
Asterisks denote viral infections/reactivations that occurred following VST infusion. Follow-up time () is as of 12/31/2014. Rows corresponding to patients who were prophylactically treated with VST are shown in grey.