Defibrotide for prophylaxis of sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) in pediatric high-risk patients: consensus guidelines from the European Society for Blood and Marrow Transplantation (EBMT)

URN zum Zitieren dieses Dokuments:

urn:nbn:de:bvb:355-epub-784287 Copy

DOI zum Zitieren dieses Dokuments:

10.5283/epub.78428 Copy

Corbacioglu, Selim ; Bajwa, Rajinder ; Antmen, Ali Bülent ; Balduzzi, Adriana ; Boelens, Jaap Jan ; Bonifazi, Francesca ; Cesaro, Simone ; Ciceri, Fabio ; Colecchia, Antonio ; Dignan, Fiona ; Kleinschmidt, Katharina ; Mahadeo, Kris M. ; Pagliuca, Antonio ; Sedlacek, Petr ; Shaw, Peter J. ; Stein, Jerry ; Szmit, Zofia ; Tambaro, Francesco ; Ince, Elif ; Verna, Marta ; Yesilipek, Akif ; Zecca, Marco ; Richardson, Paul G. ; Mohty, Mohamad ; Kalwak, Krzysztof

Lizenz: Creative Commons Namensnennung 4.0 International PDF - Veröffentlichte Version (1MB)

Veröffentlichungsdatum dieses Volltextes: 15 Jan 2026 05:21

---

Alternative Links zum Volltext:DOIVerlag

---

Diese Publikation ist Teil des DEAL-Vertrags mit Springer.

---

Details

Dokumentenart:	Artikel
Open Access Art:	DEAL (Springer)
Titel eines Journals oder einer Zeitschrift:	Bone Marrow Transplantation
Verlag:	Springer
Datum:	13 Januar 2026
Institutionen:	Medizin > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation
Identifikationsnummer:	Wert Typ 10.1038/s41409-025-02793-x DOI
Dewey-Dezimal-Klassifikation:	600 Technik, Medizin, angewandte Wissenschaften > 610 Medizin
Status:	Veröffentlicht
Begutachtet:	Ja, diese Version wurde begutachtet
An der Universität Regensburg entstanden:	Zum Teil
Dokumenten-ID:	78428

Vorschau

Bibliographische Daten exportieren

ASCII CitationAtomBibTeXData Cite XMLDataCite XML OAIDublin CoreDublin Core (LZV)EndNoteHTML CitationJSONJSON LDMETSMODSOPENAIREOpenAPCRDF+N-TriplesRDF+N3RDF+XMLReferReference ManagerSimple MetadataXMLopenCostxMetaDissPlus

---

Zusammenfassung

SOS/VOD is a life-threatening complication of hematopoietic stem cell transplantation, especially in children, with incidences reaching up to 15–20%. Despite efforts, SOS/VOD remains unpredictable with significant morbidity and mortality. High-risk criteria are clearly defined, and the pediatric EBMT diagnostic criteria have improved sensitivity, reducing treatment delays and enhancing outcomes. ...

Zusammenfassung

SOS/VOD is a life-threatening complication of hematopoietic stem cell transplantation, especially in children, with incidences reaching up to 15–20%. Despite efforts, SOS/VOD remains unpredictable with significant morbidity and mortality. High-risk criteria are clearly defined, and the pediatric EBMT diagnostic criteria have improved sensitivity, reducing treatment delays and enhancing outcomes. A meta-analysis combining retrospective and prospective studies found a risk ratio of 0.30 for SOS/VOD with defibrotide (DF) prophylaxis. Additionally, two prospective trials were conducted: the pediatric prevention trial (NCT00272948) and the Harmony Trial (NCT02851407), involving adults and children, with primary outcomes of incidence and SOS/VOD-free survival, respectively. The trials produced conflicting results regarding the effectiveness of prophylactic DF. Despite significant limitations of the Harmony trial, a direct healthcare professional communication (DHPC) from the European Medicines Agency (EMA) advised against prophylactic DF. This recommendation has serious consequences for children, especially infants, who are among the most vulnerable groups receiving HSCT. Therefore, a panel of experts issued guidelines for children at high risk for SOS/VOD, in which DF prophylaxis is considered justified. These guidelines include a weighted scoring system based on all relevant high-risk criteria to predict SOS/VOD, supporting decisions regarding the use of prophylactic DF in children.

---

---

Metadaten zuletzt geändert: 15 Jan 2026 05:21

Nur für Besitzer und Autoren: Kontrollseite des Eintrags

Downloadstatistik

Downloadstatistik

Downloadstatistik

Downloadstatistik

Downloads

Downloads im Monat während des letzten Jahres

Altmetric

Altmetric

Altmetric

Alternative Statistik (altmetrics)

Weitere Literatur (mittels CORE)

Weitere Literatur (mittels CORE)

Weitere Literatur (mittels CORE)

Weitere Literatur (mittels CORE)