Table 2.

Outcome Data

UPNSOS prophylaxis (DF mg/kg)SOS, Y/NSOS severity (moderate/severe)SOS treatment (DF dose in mg/kg/d)Cure of SOS, Y/NTime at last FU, mo post-HSCTSurvivalKarnofsky/Lansky score at last FU, %CD15+ DC at last FU, %CD3+ DC at last FU, %aGVHD (Glucksberg)cGVHD (NIH)Infection post-HSCTOther transplant-related complications post HSCT
Hep    52 Alive 100 100 88 BK Hemorrhagic cystitis 
Hep    64 Alive 100 100 100 EBV Femur head necrosis after 2 mo of steroids due to transient acute GHVD II of the liver 
NAC    16 Alive 100 100 100 EBV, rhinovirus, BK Mucositis (grade 3) 
None    Alive 40 100 100 Fungal chest infection, ADV viremia, EBV viremia Pancreatitis 
Hep/NAC    36 Alive 100 100 100 Bacterial CVL infection None 
DF 25    31 Alive 100 90 85 Rhinovirus, BCG Mucositis (grade 2), PH (d+30), gut/renal salt protein losses, renal calcification (successful surgical removal) 
DF 25 DF/steroids 24 Alive 100 98 88 EBV, BCG Hematemesis, rectal bleeding, diarrhea, early reactivation of HLH, renal impairment, arterial hypertension 
Hep DF 40 61 Alive 100 98 98 Mucositis, GI problems, Hashimoto’s thyroiditis 
NAC DF 40 paracentesis 15 Alive 100 42 None 
10 NAC    51 Alive 100 100 100 Mucositis (grade 1), CVL-associated atrial thrombus cured by surgical removal 
11 Hep DF/steroids 88 Alive 100 90 89 Mucositis (grade 2) 
12 Hep    111 Alive 100 94 92 Urinary infection Fever, mucositis (grade 2) 
13 None    45 Alive 100 100 100 None 
14 NAC    63 Alive 40 100 98 None 
15 NAC DF/steroids, paracentesis, ventilation 43 Alive 100 95 96 Mucositis (grade 2), ventilated, atelectasis left lung, urinary infection 
16 NAC DF/steroids 31 Alive 100 100 99 Recurrent CVL complications 
17 NAC    37 Alive 90 99,5 99,5 1 (oral/scalp) Vulvitis with Pseudomonas Mucositis (grade 2), engraftment syndrome, eosinophilic folliculitis of the scalp with partial effluvium, stomatitis, DD cGHVD 
18 NAC DF/steroids/paracentesis 36 Alive 100 73 30 Mucositis (grade 2) 
19 NAC, DF 25    31 Alive 100 96 87 None 
20 NAC, DF 25    18 Alive 100 58 79 Metapneumovirus, HHV-6 None 
21 Hep    14 Alive 100 100 98,5 Bacterial CVL infection Mucositis grade 3 
22 None DF 22 Alive 100 100 81 EBV viremia, rituximab 
23 Hep/DF 25    Alive 100 67 74 Mucositis (grade 3), seizure of unknown cause 
24 NAC    78 Alive 100 100 100 Mucositis (grade 1), arterial hypertension 
25 NAC    49 Alive 100 97 97 Engraftment syndrome, arthritis of 2 joints (intra-articular steroid injections) 
UPNSOS prophylaxis (DF mg/kg)SOS, Y/NSOS severity (moderate/severe)SOS treatment (DF dose in mg/kg/d)Cure of SOS, Y/NTime at last FU, mo post-HSCTSurvivalKarnofsky/Lansky score at last FU, %CD15+ DC at last FU, %CD3+ DC at last FU, %aGVHD (Glucksberg)cGVHD (NIH)Infection post-HSCTOther transplant-related complications post HSCT
Hep    52 Alive 100 100 88 BK Hemorrhagic cystitis 
Hep    64 Alive 100 100 100 EBV Femur head necrosis after 2 mo of steroids due to transient acute GHVD II of the liver 
NAC    16 Alive 100 100 100 EBV, rhinovirus, BK Mucositis (grade 3) 
None    Alive 40 100 100 Fungal chest infection, ADV viremia, EBV viremia Pancreatitis 
Hep/NAC    36 Alive 100 100 100 Bacterial CVL infection None 
DF 25    31 Alive 100 90 85 Rhinovirus, BCG Mucositis (grade 2), PH (d+30), gut/renal salt protein losses, renal calcification (successful surgical removal) 
DF 25 DF/steroids 24 Alive 100 98 88 EBV, BCG Hematemesis, rectal bleeding, diarrhea, early reactivation of HLH, renal impairment, arterial hypertension 
Hep DF 40 61 Alive 100 98 98 Mucositis, GI problems, Hashimoto’s thyroiditis 
NAC DF 40 paracentesis 15 Alive 100 42 None 
10 NAC    51 Alive 100 100 100 Mucositis (grade 1), CVL-associated atrial thrombus cured by surgical removal 
11 Hep DF/steroids 88 Alive 100 90 89 Mucositis (grade 2) 
12 Hep    111 Alive 100 94 92 Urinary infection Fever, mucositis (grade 2) 
13 None    45 Alive 100 100 100 None 
14 NAC    63 Alive 40 100 98 None 
15 NAC DF/steroids, paracentesis, ventilation 43 Alive 100 95 96 Mucositis (grade 2), ventilated, atelectasis left lung, urinary infection 
16 NAC DF/steroids 31 Alive 100 100 99 Recurrent CVL complications 
17 NAC    37 Alive 90 99,5 99,5 1 (oral/scalp) Vulvitis with Pseudomonas Mucositis (grade 2), engraftment syndrome, eosinophilic folliculitis of the scalp with partial effluvium, stomatitis, DD cGHVD 
18 NAC DF/steroids/paracentesis 36 Alive 100 73 30 Mucositis (grade 2) 
19 NAC, DF 25    31 Alive 100 96 87 None 
20 NAC, DF 25    18 Alive 100 58 79 Metapneumovirus, HHV-6 None 
21 Hep    14 Alive 100 100 98,5 Bacterial CVL infection Mucositis grade 3 
22 None DF 22 Alive 100 100 81 EBV viremia, rituximab 
23 Hep/DF 25    Alive 100 67 74 Mucositis (grade 3), seizure of unknown cause 
24 NAC    78 Alive 100 100 100 Mucositis (grade 1), arterial hypertension 
25 NAC    49 Alive 100 97 97 Engraftment syndrome, arthritis of 2 joints (intra-articular steroid injections) 

ADV, adenovirus; aGVHD, acute GVHD; BCG, bacillus Calmette-Guérin; cGVHD, chronic GVHD; CVL, central venous line; DC, donor chimerism; DD, differential diagnosis; DF, defibrotide; EBV, Epstein-Barr virus; FU, follow-up; GI, gastrointestinal; HEP, heparin; HHV-6, human herpesvirus 6; NAC, N-acetyl cysteine; NIH, National Institutes of Health; PH, pulmonary hypertension; SOS, sinusoidal obstruction syndrome.

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