Table I.
Outcomes of MAC HCT for HLH since 2002.
| Refernce | N | Predominant MAC Regimen | MRD/MUD | MMRD/MMUD | Haplo | Engrafted (%) | Mixed Chimerism (%) | Graft-Related Acute GVHD (I-IV or II-IV) | Recurrent Disease post-HCT (%) | VOD (%) | Death Prior to Day +100 | Predominant Causes of Death | Survival (years) |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Henter et al (2002) | 65 | Bu, Cy, Etop, +/- ATG | 62% | 6% | 22% | 89% | 4% | Not Specified | 5% | Not Specified | 31% | BMT Complications, Not Otherwise Specified, HLH | 62% (3-year POS) |
| Horne et al (2005) | 86 | Bu, Cy, +/- Etop, +/- ATG | 66% | 15% | 19% | 90% | 19% | 32% | 8% | Not Specified | 27% | VOD, Pneumonia, BOOP, Respiratory Failure, CMV, GVHD, Septicemia, Interstitial Pneumonia with Adenovirus and CMV, HLH | 64% (3-year POS) |
| Ouachee-Chardin et al (2006) | 48 | Bu, Cy, +/- Etop, +/- ATG | 38% | 2% | 60% | 78% | 50% | 17% | Not Specified, at least 21% | 29% | Not Specified | HLH, Infections, Not Otherwise Specified | 59% (10-year POS) |
| Eapen et al (2007) | 35 (Chediak-Higashi) | Bu, Cy, +/- Etop, +/- ATG | 60% | 29% | 11% | 94% | 23% | 52% | 23% | Not Specified, at least 3% | 26% | HLH, ARDS, VOD, Infection, Pneumonitis, GVHD | 62% (5-year POS) |
| Baker et al (2008) | 91 | Bu, Cy, Etop, +/- ATG | 59% | 39% | 0% | 91% | 10% | 41% | Not Specified, at least 8% | 18% | 35% | Infections, Interstitial Pneumonitis, VOD, Organ Failure, GVHD, Haemorrhage, HLH | 45% (5-year POS) |
| Cesaro et al (2008) | 61 | Bu, Cy, +/- Etop or Mel, +/- ATG | Not Specified | Not Specified | Not Specified | 95% | 21% | 31% | Not Specified, at least 10% | Not Specified, at least 11% | 18% | VOD, Infection, HLH | 59% (8-year POS) |
| Pachlopnik Schmid et al (2009) | 10 (Griscelli type 2) | Bu, Cy, ATG | 50% | 30% | 20% | 90% | Not Specified, at least 10% | 70% | 10% | 50% | 30% (Prior to Day 110) | VOD, Acute Respiratory Distress Secondary to Capillary Leak Syndrome or Alveolar Haemorrhage, HLH | 70% (Various) |
| Al-Ahmari et al (2010) | 11 (Griscelli type 2) | Bu, Cy, +/- Etop, +/- ATG | 73% | 27% | 0% | 100% | 33% | 9% (Grades II-IV) | 0% | 9% | 0% | Sepsis | 91% (Various) |
| Yoon et al (2010) | 19 | Bu, Cy, Etop, +/- ATG | 74% | 26% | 0% | 84% | Not Specified | 26% | 5% | 11% | 26% | Infection, Pneumonitis, Pulmonary Haemorrhage, HLH | 73% (5-year POS) |
| Marsh et al (2010b) | 14 | Bu, Cy, +/- Etop, ATG | 64% | 36% | 0% | 100% | 18% | 14% | 7% | 0% | 29% | Pulmonary Haemorrhage, ARDS, Infection, Sepsis, Multi-Organ Failure, GVHD, Organ Failure | 43% (3-year POS) |
| Ohga et al (2010) | 43 (FHLH; 1 autologous) | Bu, Cy, Etop, +/- ATG | 65% | 28% | 5% | 83% | 19% | Not Specified | 5% | Not Specified | 17% | Transplant Related Mortality, Not Otherwise Specified, HLH | 65% (10-year POS) |
MAC Myeloablative conditioning
MRD Matched related donor
MUD Matched unrelated donor
MMRD Mismatched related donor
MMUD Mismatched unrelated donor
Haplo Haploidentical
GVHD Graft-versus-host disease
HLH Haemophagocytic lymphohistiocytosis
HCT Haematopoietic cell transplantation
VOD (hepatic) Veno-occlusive disease
BOOP Bronchiolitis obliterans organizing pneumonia
Bu Busulfan
Cy Cyclophosphamide
ATG Anti-thymocyte globulin
Etop Etoposide
FHLH Familial Haemophagocytic lymphohistiocytosis
ARDS Adult respiratory distress syndrome
POS Probability of survival