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Veno-occlusive Disease in HSCT Patients: Consensus-based Recommendations for Risk Assessment, Diagnosis, and
Francesca Bonifazi1, Simona Sica2,3, Alessia Angeletti4
1IRCCS Azienda Ospedaliero-Universitaria, Istituto di Ematologia "L. e A. Seràgnoli", Bologna, Italy.
Insights
This study provides consensus recommendations for managing veno-occlusive disease (VOD) after allogeneic stem cell transplantation. Key guidelines include early diagnosis, prophylactic ursodeoxycholic acid, and prompt defibrotide treatment.
Area of Science:
- Hematology
- Transplantation Medicine
- Clinical Practice Guidelines
Background:
- Veno-occlusive disease (VOD) management varies significantly in allogeneic hematopoietic stem cell transplantation (HSCT) patients.
- Inconsistent clinical practices stem from disputes over diagnostic criteria, treatment, and prophylaxis, largely due to limited high-quality data.
- This variability impacts patient outcomes and the reliability of data collection.
Purpose of the Study:
- To reduce inconsistency in clinical care for VOD following HSCT.
- To improve patient outcomes and enhance the reliability of data collection.
- To establish evidence-based and expert-consensus recommendations for VOD management.
Main Methods:
- A collaborative effort by the Italian Society of Stem cell transplant (GISCT) was initiated.
- Recommendations were formulated by integrating available evidence and expert consensus.
- A systematic methodology aligned with US NIH and Italian National System for Guidelines was employed.
Main Results:
- Twenty-nine recommendations were approved, with 20 showing strong agreement and 9 with weak agreement.
- Twenty-six recommendations were rejected.
- Key approved recommendations emphasize early VOD diagnosis using European Society for Blood and Marrow Transplantation (EBMT) criteria and ultrasonography, prophylactic ursodeoxycholic acid, and prompt defibrotide treatment for at least 21 days.
Conclusions:
- The guidelines aim to standardize VOD management in HSCT recipients.
- Early diagnosis and prompt initiation of specific therapies are strongly advocated.
- Areas requiring further research include risk stratification and advanced diagnostic tools like elastography.
Abstract:
Variation in clinical practice affects veno-occlusive disease management, mainly in patients who undergo allogeneic hematopoietic stem cell transplantation. Disputes about diagnostic criteria, treatment, and prophylaxis, due to the lack of high-quality data, are at the base of this variability. With the aim of limiting inconsistency in clinical care, thus improving both patient outcomes and data collection reliability, the Italian Society of Stem cell transplant (Gruppo Italiano Trapianto Midollo Osseo e Terapia Cellulare) launched a collaborative effort to formulate recommendations based on integration of available evidence and expert's consensus. A systematic method, according to US National Institute of Health guidelines and Italian National System for Guidelines, was used. Twenty-nine recommendations were approved with a strong (20) or weak (9) level of agreement, while 26 were rejected. In particular, the panel pointed out the need to achieve an early diagnosis, encouraging the adoption of European Society for Blood and Marrow Transplantation criteria and the prompt use of ultrasonography. Moreover, our experts strongly recommended in favor of prophylactic use of ursodeoxycholic acid. As soon as a veno-occlusive disease diagnosis is established, treatment with defibrotide should be started for at least 21 days. A number of areas of uncertainty, particularly concerning risk stratification and use of diagnostic tools such as elastography has been identified and discussed.
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