Table 1B.
Characteristics and outcomes of 8 patients with sickle cell disease that underwent HSCT from a haploidentical, related donor with the modified Hopkins regimen.
| Transplantation Characteristics | Transplantation Outcomes | Transplant-Related Toxicity | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| No. | Age at HSCT (years) | Sex | Hemoglobin Genotype | Indications* | Prior Therapy | Donor | CD34+ Dose (×106/kg) | Duration Follow-up (months)/Current Donor T-cell Chimerism | Living Status | Neutrophil Engraftment | Infectious Complications | CMV Reactivation | aGVHD | cGVHD |
| 1 | 38 | M | SS | 8 VOC/year 3ACS/lifetime |
Hydroxyurea | Daughter (Hb AS) | 14.2 | 30 / 100% | Alive | Day +23 | E. Coli UTI | Day +19 | None | None |
| 2 | 20 | M | Sβ+-thal | 10 VOC/year | Hydroxyurea | Sister (Hb AA) | 6.0 | 23 / 90% | Alive | Day +20 | None | None | None | None |
| 3 | 21 | M | SS | 10 VOC/year TRJV = 2.7 |
Hydroxyurea | Mother (Hb AS) | 5.3 | 15 | Deceased | Day +18 | Oral HSV1 Coronavirus Influenza | Day +20 | Grade II skin Grade IV liver | Moderate - eye - liver |
| 4 | 27 | M | SS | 4 VOC/year 2 ACS/lifetime |
Hydroxyurea | Father (Hb AS) | 8.2 | 17 / 0% | Alive | Day +23 | None | None | None | None |
| 5 | 31 | M | SS | Stroke 11 VOC/year 2 ACS/last 2 years |
Hydroxyurea | Brother (Hb AA) | 4.2 | 17 / 100% | Alive | Day +22 | None | None | None | None |
| 6 | 27 | F | SS | 19 VOC/year 2 ACS/last 2 years |
Hydroxyurea | Mother (Hb AS) | 10.8 | 13 / 100% | Alive | Day +22 | Enterococcus UTI | None | None | None |
| 7 | 37 | M | SS | Stroke 3 VOC/year 2 ACS/last 2 years |
Chronic Transfusion | Mother (Hb AS) | 12.2 | 13 / 100% | Alive | Day +19 | None | None | None | None |
| 8 | 29 | F | SS | 7 VOC/year 2 ACS/lifetime |
Hydroxyurea | Mother (Hb AS) | 6.1 | 12 / 100% | Alive | Day +18 | None | None | Grade II GI | None |
*
The VOC rate is an average of the rate over the two years preceding the date of consultation for transplantation.
HSCT, hematopoietic stem cell transplant; TBI, total body irradiation; PTCy, post-transplant cyclophosphamide; CMV, cytomegalovirus; aGVHD, acute graft versus host disease; cGVHD, chronic graft versus host disease; M, male; F, female; VOC, vaso-occlusive crisis; ACS, acute chest syndrome; UTI, urinary tract infection; TRJV, tricuscpid regurgitant jet velocity; GI, gastrointestinal