Veno-occlusive Disease in HSCT Patients: Consensus-based Recommendations for Risk Assessment, Diagnosis, and Management by the GITMO Group

Bonifazi, Francesca; Sica, Simona; Angeletti, Alessia; Marktel, Sarah; Prete, Arcangelo; Iori, Anna Paola; Olivari, Diletta; Rossetti, Giulia; Bertaina, Alice; Botti, Stefano; Busca, Alessandro; Carella, Angelo Michele; Cerretti, Raffaella; Gargiulo, Gianpaolo; Grassi, Anna; Gualandi, Francesca; Irrera, Giuseppe; Milone, Giuseppe; Risitano, Antonio M.; Santarone, Stella; Vassallo, Elena; Zecca, Marco; Ciceri, Fabio; Pomponio, Giovanni

doi:10.1097/tp.0000000000003569

Cited by 7 publications

(8 citation statements)

References 59 publications

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“…Severity grading was also based on EBMT criteria based on clinical status, laboratory findings, and imaging that can distinguish between children with mild/moderate disease versus severe/very severe disease, which guided the duration of treatment of SOS/VOD and for retrospective assessment of outcomes after HSCT. Management of SOS/VOD was consistent with prevailing recommendations by EBMT and Gruppo Italiano Trapianto Midolo Osseo e Terapia Cellulare 27,28 …”

Section: Methodssupporting

confidence: 71%

Epidemiology and outcomes of pediatric transplant‐associated thrombotic microangiopathy in Hong Kong

Chan

Tak²,

Chan³

et al. 2022

Pediatric Transplantation

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Background: Transplant-associated thrombotic microangiopathy (TA-TMA) is an under-recognized yet potentially devastating complication of hematopoietic stem cell transplantation (HSCT) which had increased awareness in recent years. This report summarizes the demographics and outcomes of pediatric TA-TMA in Hong Kong. Methods: All patients aged below 18 years who underwent HSCT in the Hong Kong Children's Hospital and were diagnosed to have TA-TMA during the 2-year period from April 1, 2019 to March 31, 2021 were included.Results: A total of 73 transplants (51 allogeneic and 22 autologous) in 63 patients had been performed. Six patients (four males and two females) developed TA-TMA at a median duration of 2.5 months post-HSCT. The incidence rate was 9.52%. Of the six TA-TMA patients, five underwent allogenic one underwent autologous HSCT, respectively. Three of them were histologically proven. All four patients with cyclosporine had stopped the drug once TA-TMA was suspected. Median six doses of eculizumab were administered to five out of six patients. Three patients died (two due to fungal infection and one due to acute-on-chronic renal failure) within 3 months upon diagnosis of TA-TMA. Among three survivors, two stabilized with mild stage 2 chronic kidney disease (CKD) while the other suffered from stage 5 end-stage CKD requiring lifelong dialysis. Conclusion:In conclusion, recognition and diagnosis of TA-TMA are challenging. Early recognition and prompt administration of complement blockage with eculizumab may be beneficial in selected cases. Further prospective research studies are recommended to improve the management and outcomes of TA-TMA.

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Section: Methodssupporting

confidence: 71%

Epidemiology and outcomes of pediatric transplant‐associated thrombotic microangiopathy in Hong Kong

Chan

Tak²,

Chan³

et al. 2022

Pediatric Transplantation

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“…It is noteworthy that all patients in our series, including those not diagnosed with this complication, displayed at least one additional risk factor of SOS such as pre-existing hepatic abnormalities, active disease, rapamycin use, conditioning with TBI, use of HLA-mismatched donors, and previous allogeneic HSCT ( 1 , 2 , 6 ). Double alkylators and pre-HSCT hepatic abnormalities have been specifically associated with SOS in patients pre-treated with IO ( 3 ) and these risk factors were present in two and four patients, respectively ( Table 1 ).…”

Section: Discussionmentioning

confidence: 82%

“…Three out of seven patients (#4, #5, #6) developed all classical signs of SOS (hyperbilirubinemia, ascites, painful hepatomegaly, and weight gain) ( 1 ) a few days before engraftment, with a rapid evolution into a very severe form ( 2 ). They all received a double alkylator chemoconditioning; patient #3 also received the same chemoconditioning but died in aplasia at +11 post HSCT.…”

Section: Methods and Resultsmentioning

confidence: 99%

“…While mild SOS are underdiagnosed and spontaneously resolve, severe cases might evolve in liver dysfunction and multiple organ failure (MOF). Given the lack of pathognomonic features, several groups tried to identify clinical and laboratory criteria that define SOS ( 1 ). Several transplant, patient, and liver-related risk factors have been suggested ( 2 ) and, among them, the administration of Inotuzumab ozogamicin (IO), an anti-CD22 calicheamicin-linked monoclonal antibody approved for B-cell acute lymphoblastic leukemia (B-ALL), has been recognized as a major risk factor for both drug-induced liver injury and SOS ( 3 ).…”

Section: Introductionmentioning

confidence: 99%

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Defibrotide Prophylaxis of Sinusoidal Obstruction Syndrome in Adults Treated With Inotuzumab Ozogamicin Prior to Hematopoietic Stem Cell Transplantation

et al. 2022

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Sinusoidal Obstruction Syndrome (SOS) is a life threatening HSCT complication and it can rapidly evolve in Multiple Organ Dysfunction Syndrome, with a mortality exceeding 80%. Early treatment with defibrotide is the leading factor for efficacy. Its prophylactic use is recommended in the pediatric setting, but its value isn’t validated for adults, although factors for individual risk assessment are debated. We here present a real-world experience of Defibrotide prophylaxis in adults at very high risk of SOS. We treated with prophylactic Defibrotide and Ursodeoxycholic Acid seven patients receiving allogeneic HSCT for high risk B-ALL, previously treated with single agent Inotuzomab-Ozogamicin. They all had other high risk factors for SOS such as previous hepatotoxicity, previous allo-HSCT, double alkylating conditioning. All patients received Treosulfan-Fludarabine conditioning, Thiotepa was added in 4 patients and 4GyTBI in 2 patients. GvHD prophylaxis included post-transplant cyclophosphamide, rapamycin and mycophenolate. Donor source was PBSC. Five patients received family MMRD transplant, 1 patient a MRD transplant and 1 patient a MUD transplant. Non-severe gastrointestinal bleeding occurred in two patients requiring defibrotide temporarily discontinuation. SOS occurred in 3/7 cases within 21 days after HSCT and no late-onset SOS were diagnosed. SOS caused death in all cases. All three patients were characterized by a common pattern of very high risk factors by prior HSCT, they all received a myeloablative conditioning with Treosulfan-Thiotepa and a MMRD transplant. Defibrotide prophylaxis apparently failed to protect against the development of SOS in those patients treated with a double alkylator-based conditioning regimen, while a possible efficacy for the other high-risk patients is debatable.

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“…Insbesondere die Risikofaktoren fließen in die Beurteilung des Schweregrads mit ein. Neben den EBMT-Klassifikationen wurden weitere Empfehlungen für verschiedenen Länder und Regionen veröffentlicht (Dignan et al, 2013;Jefri et al, 2016;Bajwa et al, 2017;Mahadeo et al, 2017;Ovchinsky et al, 2017;Bonifazi et al, 2021;Cairo et al, 2020). Eine neue Klassifikation der Diagnose und Schweregrade für Kinder und Jugendliche wurde 2018 von der EBMT publiziert (Corbacioglu et al, 2018).…”

Section: Inzidenzunclassified

Von der Knochenmarktransplantation zur Intensive Care Unit – Critical Care Report

2021

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Veno-occlusive Disease in HSCT Patients: Consensus-based Recommendations for Risk Assessment, Diagnosis, and Management by the GITMO Group

Cited by 7 publications

References 59 publications

Epidemiology and outcomes of pediatric transplant‐associated thrombotic microangiopathy in Hong Kong

Epidemiology and outcomes of pediatric transplant‐associated thrombotic microangiopathy in Hong Kong

Defibrotide Prophylaxis of Sinusoidal Obstruction Syndrome in Adults Treated With Inotuzumab Ozogamicin Prior to Hematopoietic Stem Cell Transplantation

Von der Knochenmarktransplantation zur Intensive Care Unit – Critical Care Report

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