Detailed patient and transplant characteristics (N = 25)
| No./year of HCT . | Age at dx, mo . | Age at HCT, mo . | Interval between dx and HCT, mo . | Pretransplant infections and medical issues . | Donor . | Stem cell source . | Conditioning and GvHD prophylaxis . | Acute GvHD . | Significant complications during and after HCT . | Outcome . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1/1995 | 4.9 | 7.0 | 2.1 | Disseminated CMV infection (blood, liver, stools, NPS, urine). | MFD (paternal uncle) | BM | Oral Bu 16 mg/kg (Bu AUC 1837 μmol × min). | None | Pneumonitis required mechanical ventilation. | Died of CMV pneumonitis (postmortem) at day +17. |
| Poliovirus and rotavirus (stools). | Cy 200 mg/kg. | AKI, seizures, toxic epidermal necrolysis (skin biopsy proven). | ||||||||
| Haemophilus influenzae (NPS). Maternal T-cell engraftment (skin biopsy proven). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA. | ||||||||||
| 2/1997 | At birth | 0.9 | 0.8 | Staphylococcus aureus septicemia. | MFD (great-granduncle) | BM | Oral Bu 16 mg/kg (no Bu pK). | None | Engraftment pneumonitis required mechanical ventilation and responded IST (treated with neb budesonide, neb Ig, systemic steroid, ATG, anti-TNF monoclonal antibody). | Secondary autologous reconstitution at day +76. |
| Cy 200 mg/kg. Rabbit ATG 2.2 mg/kg. | Severe hypertension with seizures. | Second HCT using a different 7/8 MMFD at 5.6 mo of age. | ||||||||
| CSA/MTX. | CMV viremia. Staphylococcus epidermidis bacteremia. | Died of interstitial pneumonitis at day +88 postsecond HCT. | ||||||||
| Diagnosed as pyloric stenosis at day +56 and underwent pyloromyotomy on day +69. | ||||||||||
| 3/1997 | 4.0 | 5.8 | 1.8 | PCP (BAL). | MSD | BM | Oral Bu 16 mg/kg (no Bu pK). | None | Worsening pretransplant respiratory dysfunction from day +9 and required mechanical ventilation from day +17. | Died of parainfluenza virus 3 pneumonitis at day +19. |
| Candida perianal ulcers. | Cy 200 mg/kg. | NPS was positive for parainfluenza virus 3 and rhinovirus. | ||||||||
| Congenital retinal dystrophy. | No serotherapy. | Treated with neb budesonide, neb IVIg, systemic steroid, ATG, and neb and IV ribavirin. | ||||||||
| CSA/MTX. | ||||||||||
| 4/2003 | 4.7 | 6.2 | 1.5 | PCP (BAL). | 7/8 MMUD | CB | Flu 150 mg/m2. Melph 140 mg/m2. Alemtuzumab 1 mg/kg. | None | Staphylococcus epidermidis bacteremia. | Alive |
| Fungal (hyphae on BAL). | CSA/steroid. | CMV pneumonitis rotavirus. | ||||||||
| Poliovirus (gut and NPS). | ||||||||||
| 5/2008 | 8.4 | 11.3 | 2.9 | PCP (BAL). | MFD | BM | Treo 36 mg/m2. | Grade 1 skin | Pneumonitis. | Cryopreserved marrow top-up at day +54 for slipping chimerism. |
| Norovirus type 2 enteropathy. | Cy 200 mg/kg. | Capillary leakage syndrome. | Second conditioned HCT using the same donor at day +186. | |||||||
| HHV6 viremia. | No serotherapy. CSA/MMF. | Diagnosed to have pelvic ureteric junction obstruction at 2 y of age following urosepsis. | Died of urosepsis 3.8 y postsecond HCT. | |||||||
| Maternal T-cell engraftment. | Had stenting done. | |||||||||
| Seizure with neurodevelopmental delay and absent corpus collusum. | Had sterile liver nodules 2.8 y of age. | |||||||||
| Operated gut malrotation. | Required ventricular peritoneal shunt at 3 y of age. | |||||||||
| 6/2008 | 15.3 | 17.7 | 2.4 | CMV (NPS). | MSD | BM | Treo 42 mg/m2. | Grade 1 skin | CMV viremia. | Died of pneumonitis with multiorgan failure at day +261. |
| Norovirus type 2 enteropathy. RSV and parainfluenza virus 3 (NPS). | Cy 200 mg/kg. | Adenovirus (stools). | Post mortem examination revealed positive HHV6 in lung and liver. | |||||||
| Probable autoimmune hepatitis (isolated raised hepatic transaminases). | No serotherapy. CSA/MMF. | Developed respiratory failure required mechanical ventilation at 8 mo post-HCT. | ||||||||
| 7/2011 | 7.8 | 9.8 | 2.0 | PCP. | MRD | PBSC | Treo 36 g/m2. | Grade 1 skin | Engraftment pneumonitis (treated with neb budesonide). | Alive |
| Chronic diarrhea (normal gut biopsy and negative pathogens). | Flu 150 mg/m2. | |||||||||
| Alemtuzumab 1 mg/kg. | ||||||||||
| CSA/MMF. | ||||||||||
| 8*/2012 | 89.9 | 93.3 | 3.5 | Polyarticular juvenile idiopathic arthritis since 14 mo of age, treated with steroid, etanercept and infliximab. | MUD | PBSC | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus (blood, stools, and NPS). | Alive |
| Macrophage-activating syndrome at 7 y of age, treated with steroid and CSA. Mycobacterium avium-M. intracellulare (sputum and BAL). | Flu 150 mg/m2. | CMV viremia. | ||||||||
| H. influenzae (BAL). | Alemtuzumab 1 mg/kg. | Micrococcus luteus bacteremia. | ||||||||
| CMV (BAL). | CSA/MMF. | |||||||||
| HHV7 (blood). | ||||||||||
| Norovirus and sapovirus (stools). | ||||||||||
| Right middle lobe bronchiectasis. | ||||||||||
| 9*/2012 | 22.7 | 27.4 | 4.8 | Macrophage-activating syndrome at 2 y old, treated with steroid and CSA. | 9/10 A-MMUD | PBSCs | Treo 42 g/m2. | Grade 2 skin | Disseminated adenovirus (blood, gut, NPS). | Alive |
| Bronchiectasis. | Flu 150 mg/m2. | CMV viremia. | ||||||||
| Norovirus enteropathy. | Alemtuzumab 1 mg/kg. | |||||||||
| CMV (BAL). | CSA/MMF | |||||||||
| 10/2015 | 8.0 | 16.2 | 8.2 | PCP. | 9/10 C-MMUD | BM | Treo 42 g/m2. | Grade 1 skin | S. epidermidis bacteremia. | Alive |
| H. influenzae (BAL). | Flu 150 mg/m2. | Immune reconstitution . abscess at day +141. | ||||||||
| Pseudomonas aeruginosa (BAL). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF | ||||||||||
| 11/2015 | 6.0 | 29.4 | 23.4 | Diagnosed at 6 mo because older sibling died at 6 y. | Maternal HID | TCRαβ/CD19-depleted PBSCs | Treo 42 g/m2. | Grade 2 skin | Disseminated adenovirus (blood, stool, NPS); received adenovirus CTL. | Alive |
| Disseminated adenovirus (BAL, blood stools). | Flu 150 mg/m2. | HHV6 viremia. | ||||||||
| Multiple gut viruses (norovirus and adenovirus). | Thio 10 mg/kg. | Parainfluenza virus 1. | ||||||||
| ATG 15 mg/kg. | Escherichia coli urinary tract infection. | |||||||||
| RTX 200 mg/mg. | ||||||||||
| CSA. | ||||||||||
| 12*/2015 | 7.6 | 15.7 | 8.1 | Enterovirus hepatitis (biopsy proven). | 9/10 DQ-MMUD | PBSCs | Treo 42 g/m2. | Grade 2 skin | None | Alive |
| PN-dependent enteropathy with multiple gut viruses (enterovirus and sapovirus). | Flu 150 mg/m2. | Stopped PN at day +135 post-HCT. | ||||||||
| Streptococcus oralis bacteremia. | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF. | ||||||||||
| 13*/2015 | 6.6 | 16.4 | 9.8 | PCP. Enterovirus viremia and hepatitis (biopsy proven). | 9/10 DQ-MMUD | PBSCs | Treo 42 g/m2. | Grade 1 skin | None | Alive |
| PN-dependent enteropathy with multiple gut viruses (enterovirus and sapovirus). E. coli and alpha hemolytic Streptococcus bacteremia. | Flu 150 mg/m2. | Stopped PN at day +110 post-HCT. | ||||||||
| Alemtuzumab 1 mg/kg. | ||||||||||
| CSA/MMF. | ||||||||||
| 14/2015 | 6.5 | 21.4 | 14.9 | PCP. | Paternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | None | Enterovirus meningitis with communicating hydrocephalus on day +56. | Alive |
| Multiple gut viruses (norovirus, enterovirus). | Flu 150 mg/m2. | |||||||||
| Thio 10 mg/kg. | ||||||||||
| RTX 200 mg/m2. | ||||||||||
| ATG 15 mg/kg. | ||||||||||
| CSA. | ||||||||||
| 15/2016 | At birth | 47.7 | 47.7 | CMV viremia. | MUD | PBSCs | Treo 42 g/m2. | None | Disseminated adenovirus (blood, eye swab). | Alive |
| Disseminated parechovirus (blood, stool). | Flu 150 mg/m2. | CMV viremia. | ||||||||
| Sapovirus (stool). | Alemtuzumab 1 mg/kg. | |||||||||
| Nontuberculous mycobacteria of lung (biopsy proven). | CSA/MMF. | |||||||||
| E. coli urinary tract infection. | ||||||||||
| Osteopenic fracture of right tibia and fibula. | ||||||||||
| 16/2016 | At birth | 9.6 | 9.6 | Parainfluenza virus 3 (BAL). | MUD | PBSCs | Treo 42 g/m2. | None | None | Alive |
| Streptococcus pneumoniae (BAL). | Flu 150 mg/m2. | |||||||||
| Enterovirus (stool). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF. | ||||||||||
| 17/2017 | 17 | 31 | 14 | Multiple gut viruses (adenovirus, sapovirus, enterovirus, norovirus). | Maternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus with pericardial effusion requiring pericardial window. | Alive |
| Multiple respiratory viruses (parainfluenza virus 3, adenovirus, RSV). | Add-back T cells. | Flu 150 mg/m2. | ||||||||
| HHV6 viremia. | Thio 10 mg/kg. | |||||||||
| S. pneumoniae (BAL). | RTX 200 mg/m2 . | |||||||||
| ATG 15 mg/kg. | ||||||||||
| No GvHD prophylaxis. | ||||||||||
| 18/2017 | 6 | 60.7 | 54.7 | PCP. | Paternal HID | TCRαβ/CD19-depleted PBSC. | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus (blood, stool, NPS). | Alive |
| Severe malnutrition. | Add-back T cells. | Flu 150 mg/m2. | Stopped PN at day +67 post-HCT. | |||||||
| PN-dependent enteropathy with multiple gut viruses (norovirus, adenovirus, enterovirus). | Thio 10 mg/kg. | |||||||||
| HHV6 viremia. | RTX 200 mg/m2. | |||||||||
| Norovirus and adenovirus enteropathy. | ATG 15 mg/kg. | |||||||||
| Presumed fungal splenic abscess. | No GvHD prophylaxis. | |||||||||
| Multiple osteopenic fractures secondary to vitamin D deficiency. | ||||||||||
| 19*/2017 | 13.2 | 81.7 | 68.4 | PCP. | MUD | PBSCs | Treo 42 g/m2. | None | Disseminated adenovirus (blood, stool, NPS). | Alive |
| Norovirus (gut). | Flu 150 mg/m2. | |||||||||
| HHV6 viremia. | Alemtuzumab 1 mg/kg. | |||||||||
| 20/2017 | 4.6 | 12.3 | 7.7 | HHV 6 (blood and CSF). Adenovirus (blood and stool). Coxsackievirus A type 6 (stool, NPS). | Paternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus (blood, stool). | Alive |
| Norovirus (stool). | Flu 150 mg/m2. | HHV6 viremia. | ||||||||
| RSV and parainfluenza virus 2 and 3 on NPS. | Thio 10 mg/kg. | Encephalopathy of unknown etiology (normal CSF and MRI brain). | ||||||||
| RTX 200 mg/m2. ATG 15 mg/kg. | Full neurological recovery. | |||||||||
| No GvHD prophylaxis. | ||||||||||
| 21*/2017 | At birth | 62.7 | 62.7 | Severe malnutrition. | MUD | PBSCs | Treo 42 g/m2. | None | HHV6 viremia. | Alive |
| PN-dependent enteropathy with multiple gut viruses (norovirus, parechovirus). | Flu 150 mg/m2. | Stopped PN at day +59 post-HCT. | ||||||||
| Parainfluenza virus 4. | Alemtuzumab 1 mg/kg. | |||||||||
| HHV6 viremia. | CSA/MMF. | |||||||||
| 22/2017 | 24 | 73.6 | 49.6 | Severe malnutrition. | Paternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | Grade 1 skin | PN-dependent gut failure. | Received second HCT for secondary aplasia. |
| Disseminated adenovirus (blood, BAL, stools). | Add-back T cells. | Flu 150 mg/m2. | Disseminated adenovirus. | Died of cerebral hemorrhage postsecond HCT. | ||||||
| Disseminated CMV (blood, BAL). | Thio 10 mg/kg. | RSV pneumonia. | ||||||||
| EBV in BAL. | RTX 200 mg/m2. | HHV6 viremia. | ||||||||
| HHV6 viremia. | ATG 15 mg/kg. | |||||||||
| RSV (NPS). | No GvHD prophylaxis. | |||||||||
| Multiple gut viruses (adenovirus, enterovirus, sapovirus, astrovirus, norovirus). | ||||||||||
| 23/2018 | 6.0 | 22.2 | 16.7 | HHV6 viremia. | MUD | PBSCs | Treo 42 g/m2. | None | HHV6 viremia. | Alive |
| Norovirus (stool). | Flu 150 mg/m2. | |||||||||
| RSV and parainfluenza virus 1 (NPS). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF. | ||||||||||
| 24/2018 | 5.0 | 78.8 | 73.8 | PCP. | 9/10 A-MMUD | PBSCs | Treo 42 g/m2. | Grade 2 skin | HHV6 viremia. | Alive |
| RSV pneumonia. | Flu 150 mg/m2. | PN-dependent viral enteropathy on gut biopsy; no evidence of gut GvHD on gut biopsy. | Stopped PN at day +641 post-HCT. | |||||||
| PN-dependent enteropathy with multiple gut viruses (adenovirus, norovirus, astrovirus). | Alemtuzumab 1 mg/kg. | Slow immune reconstitution secondary steroid-dependent skin acute GvHD. | ||||||||
| Candida esophagitis. | CSA/MMF. | |||||||||
| Disseminated BCG at 3 y of age. | ||||||||||
| 25/2018 | 6.0 | 22.8 | 16.8 | PCP. | Maternal HID | TCRαβ/CD19-depleted PBSCs | Treo 42 g/m2. | Grade 2 skin | None | Alive |
| Chronic diarrhea (Salmonella spp. and norovirus). | Flu 150 mg/m2. | |||||||||
| Thio 10 mg/kg. | ||||||||||
| RTX 200 mg/m2. | ||||||||||
| ATG 15 mg/kg. | ||||||||||
| No GvHD prophylaxis. |
| No./year of HCT . | Age at dx, mo . | Age at HCT, mo . | Interval between dx and HCT, mo . | Pretransplant infections and medical issues . | Donor . | Stem cell source . | Conditioning and GvHD prophylaxis . | Acute GvHD . | Significant complications during and after HCT . | Outcome . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1/1995 | 4.9 | 7.0 | 2.1 | Disseminated CMV infection (blood, liver, stools, NPS, urine). | MFD (paternal uncle) | BM | Oral Bu 16 mg/kg (Bu AUC 1837 μmol × min). | None | Pneumonitis required mechanical ventilation. | Died of CMV pneumonitis (postmortem) at day +17. |
| Poliovirus and rotavirus (stools). | Cy 200 mg/kg. | AKI, seizures, toxic epidermal necrolysis (skin biopsy proven). | ||||||||
| Haemophilus influenzae (NPS). Maternal T-cell engraftment (skin biopsy proven). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA. | ||||||||||
| 2/1997 | At birth | 0.9 | 0.8 | Staphylococcus aureus septicemia. | MFD (great-granduncle) | BM | Oral Bu 16 mg/kg (no Bu pK). | None | Engraftment pneumonitis required mechanical ventilation and responded IST (treated with neb budesonide, neb Ig, systemic steroid, ATG, anti-TNF monoclonal antibody). | Secondary autologous reconstitution at day +76. |
| Cy 200 mg/kg. Rabbit ATG 2.2 mg/kg. | Severe hypertension with seizures. | Second HCT using a different 7/8 MMFD at 5.6 mo of age. | ||||||||
| CSA/MTX. | CMV viremia. Staphylococcus epidermidis bacteremia. | Died of interstitial pneumonitis at day +88 postsecond HCT. | ||||||||
| Diagnosed as pyloric stenosis at day +56 and underwent pyloromyotomy on day +69. | ||||||||||
| 3/1997 | 4.0 | 5.8 | 1.8 | PCP (BAL). | MSD | BM | Oral Bu 16 mg/kg (no Bu pK). | None | Worsening pretransplant respiratory dysfunction from day +9 and required mechanical ventilation from day +17. | Died of parainfluenza virus 3 pneumonitis at day +19. |
| Candida perianal ulcers. | Cy 200 mg/kg. | NPS was positive for parainfluenza virus 3 and rhinovirus. | ||||||||
| Congenital retinal dystrophy. | No serotherapy. | Treated with neb budesonide, neb IVIg, systemic steroid, ATG, and neb and IV ribavirin. | ||||||||
| CSA/MTX. | ||||||||||
| 4/2003 | 4.7 | 6.2 | 1.5 | PCP (BAL). | 7/8 MMUD | CB | Flu 150 mg/m2. Melph 140 mg/m2. Alemtuzumab 1 mg/kg. | None | Staphylococcus epidermidis bacteremia. | Alive |
| Fungal (hyphae on BAL). | CSA/steroid. | CMV pneumonitis rotavirus. | ||||||||
| Poliovirus (gut and NPS). | ||||||||||
| 5/2008 | 8.4 | 11.3 | 2.9 | PCP (BAL). | MFD | BM | Treo 36 mg/m2. | Grade 1 skin | Pneumonitis. | Cryopreserved marrow top-up at day +54 for slipping chimerism. |
| Norovirus type 2 enteropathy. | Cy 200 mg/kg. | Capillary leakage syndrome. | Second conditioned HCT using the same donor at day +186. | |||||||
| HHV6 viremia. | No serotherapy. CSA/MMF. | Diagnosed to have pelvic ureteric junction obstruction at 2 y of age following urosepsis. | Died of urosepsis 3.8 y postsecond HCT. | |||||||
| Maternal T-cell engraftment. | Had stenting done. | |||||||||
| Seizure with neurodevelopmental delay and absent corpus collusum. | Had sterile liver nodules 2.8 y of age. | |||||||||
| Operated gut malrotation. | Required ventricular peritoneal shunt at 3 y of age. | |||||||||
| 6/2008 | 15.3 | 17.7 | 2.4 | CMV (NPS). | MSD | BM | Treo 42 mg/m2. | Grade 1 skin | CMV viremia. | Died of pneumonitis with multiorgan failure at day +261. |
| Norovirus type 2 enteropathy. RSV and parainfluenza virus 3 (NPS). | Cy 200 mg/kg. | Adenovirus (stools). | Post mortem examination revealed positive HHV6 in lung and liver. | |||||||
| Probable autoimmune hepatitis (isolated raised hepatic transaminases). | No serotherapy. CSA/MMF. | Developed respiratory failure required mechanical ventilation at 8 mo post-HCT. | ||||||||
| 7/2011 | 7.8 | 9.8 | 2.0 | PCP. | MRD | PBSC | Treo 36 g/m2. | Grade 1 skin | Engraftment pneumonitis (treated with neb budesonide). | Alive |
| Chronic diarrhea (normal gut biopsy and negative pathogens). | Flu 150 mg/m2. | |||||||||
| Alemtuzumab 1 mg/kg. | ||||||||||
| CSA/MMF. | ||||||||||
| 8*/2012 | 89.9 | 93.3 | 3.5 | Polyarticular juvenile idiopathic arthritis since 14 mo of age, treated with steroid, etanercept and infliximab. | MUD | PBSC | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus (blood, stools, and NPS). | Alive |
| Macrophage-activating syndrome at 7 y of age, treated with steroid and CSA. Mycobacterium avium-M. intracellulare (sputum and BAL). | Flu 150 mg/m2. | CMV viremia. | ||||||||
| H. influenzae (BAL). | Alemtuzumab 1 mg/kg. | Micrococcus luteus bacteremia. | ||||||||
| CMV (BAL). | CSA/MMF. | |||||||||
| HHV7 (blood). | ||||||||||
| Norovirus and sapovirus (stools). | ||||||||||
| Right middle lobe bronchiectasis. | ||||||||||
| 9*/2012 | 22.7 | 27.4 | 4.8 | Macrophage-activating syndrome at 2 y old, treated with steroid and CSA. | 9/10 A-MMUD | PBSCs | Treo 42 g/m2. | Grade 2 skin | Disseminated adenovirus (blood, gut, NPS). | Alive |
| Bronchiectasis. | Flu 150 mg/m2. | CMV viremia. | ||||||||
| Norovirus enteropathy. | Alemtuzumab 1 mg/kg. | |||||||||
| CMV (BAL). | CSA/MMF | |||||||||
| 10/2015 | 8.0 | 16.2 | 8.2 | PCP. | 9/10 C-MMUD | BM | Treo 42 g/m2. | Grade 1 skin | S. epidermidis bacteremia. | Alive |
| H. influenzae (BAL). | Flu 150 mg/m2. | Immune reconstitution . abscess at day +141. | ||||||||
| Pseudomonas aeruginosa (BAL). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF | ||||||||||
| 11/2015 | 6.0 | 29.4 | 23.4 | Diagnosed at 6 mo because older sibling died at 6 y. | Maternal HID | TCRαβ/CD19-depleted PBSCs | Treo 42 g/m2. | Grade 2 skin | Disseminated adenovirus (blood, stool, NPS); received adenovirus CTL. | Alive |
| Disseminated adenovirus (BAL, blood stools). | Flu 150 mg/m2. | HHV6 viremia. | ||||||||
| Multiple gut viruses (norovirus and adenovirus). | Thio 10 mg/kg. | Parainfluenza virus 1. | ||||||||
| ATG 15 mg/kg. | Escherichia coli urinary tract infection. | |||||||||
| RTX 200 mg/mg. | ||||||||||
| CSA. | ||||||||||
| 12*/2015 | 7.6 | 15.7 | 8.1 | Enterovirus hepatitis (biopsy proven). | 9/10 DQ-MMUD | PBSCs | Treo 42 g/m2. | Grade 2 skin | None | Alive |
| PN-dependent enteropathy with multiple gut viruses (enterovirus and sapovirus). | Flu 150 mg/m2. | Stopped PN at day +135 post-HCT. | ||||||||
| Streptococcus oralis bacteremia. | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF. | ||||||||||
| 13*/2015 | 6.6 | 16.4 | 9.8 | PCP. Enterovirus viremia and hepatitis (biopsy proven). | 9/10 DQ-MMUD | PBSCs | Treo 42 g/m2. | Grade 1 skin | None | Alive |
| PN-dependent enteropathy with multiple gut viruses (enterovirus and sapovirus). E. coli and alpha hemolytic Streptococcus bacteremia. | Flu 150 mg/m2. | Stopped PN at day +110 post-HCT. | ||||||||
| Alemtuzumab 1 mg/kg. | ||||||||||
| CSA/MMF. | ||||||||||
| 14/2015 | 6.5 | 21.4 | 14.9 | PCP. | Paternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | None | Enterovirus meningitis with communicating hydrocephalus on day +56. | Alive |
| Multiple gut viruses (norovirus, enterovirus). | Flu 150 mg/m2. | |||||||||
| Thio 10 mg/kg. | ||||||||||
| RTX 200 mg/m2. | ||||||||||
| ATG 15 mg/kg. | ||||||||||
| CSA. | ||||||||||
| 15/2016 | At birth | 47.7 | 47.7 | CMV viremia. | MUD | PBSCs | Treo 42 g/m2. | None | Disseminated adenovirus (blood, eye swab). | Alive |
| Disseminated parechovirus (blood, stool). | Flu 150 mg/m2. | CMV viremia. | ||||||||
| Sapovirus (stool). | Alemtuzumab 1 mg/kg. | |||||||||
| Nontuberculous mycobacteria of lung (biopsy proven). | CSA/MMF. | |||||||||
| E. coli urinary tract infection. | ||||||||||
| Osteopenic fracture of right tibia and fibula. | ||||||||||
| 16/2016 | At birth | 9.6 | 9.6 | Parainfluenza virus 3 (BAL). | MUD | PBSCs | Treo 42 g/m2. | None | None | Alive |
| Streptococcus pneumoniae (BAL). | Flu 150 mg/m2. | |||||||||
| Enterovirus (stool). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF. | ||||||||||
| 17/2017 | 17 | 31 | 14 | Multiple gut viruses (adenovirus, sapovirus, enterovirus, norovirus). | Maternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus with pericardial effusion requiring pericardial window. | Alive |
| Multiple respiratory viruses (parainfluenza virus 3, adenovirus, RSV). | Add-back T cells. | Flu 150 mg/m2. | ||||||||
| HHV6 viremia. | Thio 10 mg/kg. | |||||||||
| S. pneumoniae (BAL). | RTX 200 mg/m2 . | |||||||||
| ATG 15 mg/kg. | ||||||||||
| No GvHD prophylaxis. | ||||||||||
| 18/2017 | 6 | 60.7 | 54.7 | PCP. | Paternal HID | TCRαβ/CD19-depleted PBSC. | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus (blood, stool, NPS). | Alive |
| Severe malnutrition. | Add-back T cells. | Flu 150 mg/m2. | Stopped PN at day +67 post-HCT. | |||||||
| PN-dependent enteropathy with multiple gut viruses (norovirus, adenovirus, enterovirus). | Thio 10 mg/kg. | |||||||||
| HHV6 viremia. | RTX 200 mg/m2. | |||||||||
| Norovirus and adenovirus enteropathy. | ATG 15 mg/kg. | |||||||||
| Presumed fungal splenic abscess. | No GvHD prophylaxis. | |||||||||
| Multiple osteopenic fractures secondary to vitamin D deficiency. | ||||||||||
| 19*/2017 | 13.2 | 81.7 | 68.4 | PCP. | MUD | PBSCs | Treo 42 g/m2. | None | Disseminated adenovirus (blood, stool, NPS). | Alive |
| Norovirus (gut). | Flu 150 mg/m2. | |||||||||
| HHV6 viremia. | Alemtuzumab 1 mg/kg. | |||||||||
| 20/2017 | 4.6 | 12.3 | 7.7 | HHV 6 (blood and CSF). Adenovirus (blood and stool). Coxsackievirus A type 6 (stool, NPS). | Paternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | Grade 1 skin | Disseminated adenovirus (blood, stool). | Alive |
| Norovirus (stool). | Flu 150 mg/m2. | HHV6 viremia. | ||||||||
| RSV and parainfluenza virus 2 and 3 on NPS. | Thio 10 mg/kg. | Encephalopathy of unknown etiology (normal CSF and MRI brain). | ||||||||
| RTX 200 mg/m2. ATG 15 mg/kg. | Full neurological recovery. | |||||||||
| No GvHD prophylaxis. | ||||||||||
| 21*/2017 | At birth | 62.7 | 62.7 | Severe malnutrition. | MUD | PBSCs | Treo 42 g/m2. | None | HHV6 viremia. | Alive |
| PN-dependent enteropathy with multiple gut viruses (norovirus, parechovirus). | Flu 150 mg/m2. | Stopped PN at day +59 post-HCT. | ||||||||
| Parainfluenza virus 4. | Alemtuzumab 1 mg/kg. | |||||||||
| HHV6 viremia. | CSA/MMF. | |||||||||
| 22/2017 | 24 | 73.6 | 49.6 | Severe malnutrition. | Paternal HID | TCRαβ/CD19-depleted PBSCs. | Treo 42 g/m2. | Grade 1 skin | PN-dependent gut failure. | Received second HCT for secondary aplasia. |
| Disseminated adenovirus (blood, BAL, stools). | Add-back T cells. | Flu 150 mg/m2. | Disseminated adenovirus. | Died of cerebral hemorrhage postsecond HCT. | ||||||
| Disseminated CMV (blood, BAL). | Thio 10 mg/kg. | RSV pneumonia. | ||||||||
| EBV in BAL. | RTX 200 mg/m2. | HHV6 viremia. | ||||||||
| HHV6 viremia. | ATG 15 mg/kg. | |||||||||
| RSV (NPS). | No GvHD prophylaxis. | |||||||||
| Multiple gut viruses (adenovirus, enterovirus, sapovirus, astrovirus, norovirus). | ||||||||||
| 23/2018 | 6.0 | 22.2 | 16.7 | HHV6 viremia. | MUD | PBSCs | Treo 42 g/m2. | None | HHV6 viremia. | Alive |
| Norovirus (stool). | Flu 150 mg/m2. | |||||||||
| RSV and parainfluenza virus 1 (NPS). | Alemtuzumab 1 mg/kg. | |||||||||
| CSA/MMF. | ||||||||||
| 24/2018 | 5.0 | 78.8 | 73.8 | PCP. | 9/10 A-MMUD | PBSCs | Treo 42 g/m2. | Grade 2 skin | HHV6 viremia. | Alive |
| RSV pneumonia. | Flu 150 mg/m2. | PN-dependent viral enteropathy on gut biopsy; no evidence of gut GvHD on gut biopsy. | Stopped PN at day +641 post-HCT. | |||||||
| PN-dependent enteropathy with multiple gut viruses (adenovirus, norovirus, astrovirus). | Alemtuzumab 1 mg/kg. | Slow immune reconstitution secondary steroid-dependent skin acute GvHD. | ||||||||
| Candida esophagitis. | CSA/MMF. | |||||||||
| Disseminated BCG at 3 y of age. | ||||||||||
| 25/2018 | 6.0 | 22.8 | 16.8 | PCP. | Maternal HID | TCRαβ/CD19-depleted PBSCs | Treo 42 g/m2. | Grade 2 skin | None | Alive |
| Chronic diarrhea (Salmonella spp. and norovirus). | Flu 150 mg/m2. | |||||||||
| Thio 10 mg/kg. | ||||||||||
| RTX 200 mg/m2. | ||||||||||
| ATG 15 mg/kg. | ||||||||||
| No GvHD prophylaxis. |
AKI, acute kidney injury; AUC, area under the curve; BAL, bronchoalveolar lavage; BCG, bacille Calmette-Guerin; BM, bone marrow; Bu, busulfan; CB, cord blood; CSF, cerebrospinal fluid; CTL, cytotoxic T cells; Cy, cyclophosphamide; dx, diagnosis; Flu, fludarabine; IST, immunosuppressive therapy; Melph, melphalan; MMFD, mismatched family donor; MRI, magnetic resonance imaging; neb, nebulized; NPS, nasopharyngeal specimen; pK, pharmacokinetics; PN, parenteral nutrition; RSV, respiratory syncytial virus; RTX, rituximab; Thio, thiotepa; Treo, treosulfan; TNF, tumor necrosis factor.