Table 3.
Paroxysmal nocturnal hemoglobinuria
| PRE-HCT CHARACTERISTICS | PATIENT #8 | PATIENT #9 | PATIENT #10 | PATIENT #11 |
|---|---|---|---|---|
| Age at diagnosis/ at HCT (yr) | 15.4 / 15.9 | 19 / 22.1 | 14.5 / 17.6 | 14 / 14.9 |
| Patient sex | Female | Female | Female | Male |
| GP-1 flow cytometry | GP1-deficient neutrophils (57–60%), monocytes (62%), erythrocytes (1%) | Normal post 1st-HCT | GP1-deficient neutrophils (82%), monocytes (79.2%), and erythrocytes (5.7%) | GP1-deficient neutrophils (27.7%), monocytes (77.6%), and erythrocytes (54.1%) |
| History of eculizumab | NO | NO | Yes; initiated 3 days prior to start of conditioning and continued through engraftment | Yes; initiated 11 months prior to HCT and continued through engraftment |
| Augmented HCT/CI | 1 | 8 | 1 | 0 |
| Peripheral blood counts | ||||
| ANC (cells/µL) | 720 | 700 | 380 | 560 |
| Hematocrit | 25% | 23% | 22% | 32% |
| Platelet count (cells/µL) | 13,000 | 33,000 | 13,000 | 179,000 |
| Transfusion dependent | Yes (pRBCs and platelets) | Yes (pRBCs and platelets) | Yes (pRBCs and platelets) | No |
| Bone marrow evaluation | ||||
| Cellularity | 10%-20% | 70% | 70% | 70% |
| Cytogenetics | Normal | Normal | Normal | Normal |
| Indication for HCT | Cytopenias, G-CSF dependent, pRBC and platelet transfusion dependent | Cytopenias following 1st HCT, pRBC and platelet transfusion dependent. | Cytopenias, pRBC and platelet transfusion dependent | Failed Eculizumab (profound fatigue, continued hemolysis) |
| HCT Characteristics and Outcomes | ||||
| Donor/Stem cell source | MURD/PBSC | MURD/marrow | A-Allele MMURD/PBSC | MRD/BM |
| Follow-up (yr) | Alive, 6.5 | Died GVHD, 0.4 | Alive, 2.3 | Alive, 1.4 |
| Donor Chimerism (CD3+/CD33+)† | 100%/100% | 100%/100% | 97%/100% | 95%/100% |
| GVHD§ | ||||
| Acute/Chronic (Yes/No) | No/No | Yes/No | No/Yes | No/No |
| Day Immune Suppression Stopped | 160 | Died on | 423 | 513 |
| Peripheral blood counts† | ||||
| ANC (cells/µL) | 3300 | 3965 | 4400 | 3460 |
| Hematocrit | 40.9% | 27.9% | 34.5% | 42% |
| Platelet Count (cells/µL) | 289,000 | 53,000 | 275,000 | 231,000 |
| Bone marrow evaluation† | ||||
| Cellularity | N/A | 20% | 70% | 50% |
| Cytogenetics | Normal | Normal | Normal | Normal |
| Disease response | Normal GP-1 expression | Normal GP-1 expression | Normal GP-1 expression | Normal GP-1 expression |
†
at time point last studied
§
Acute grade II-IV GVHD, delayed acute GVHD or NIH chronic GVHD
Abbreviations: HCT, hematopoietic cell transplantation; HCT-CI, hematopoietic cell transplantation comorbidity index; MRD, matched related donor; MURD, matched unrelated donor; MM, mismatched; N/A, not available; PBSC, peripheral blood stem cells; pRBC, packed red blood cell; R, recipient; yr, years