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Improved detection of sinusoidal obstructive syndrome using pediatric-AYA diagnostic criteria and severity grading
D Ragoonanan1, S J Khazal2, J Wang3
1Department of Pediatrics, Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, 1515 Holcombe Boulevard, Houston, TX, 77030, USA. DRagoonanan@mdanderson.org.
Insights
New criteria for sinusoidal obstructive syndrome (SOS) in pediatric patients diagnose cases earlier and more frequently. Applying the pEBMT guidelines in the US may improve timely treatment for hematopoietic cell transplantation recipients.
Area of Science:
- Hematology
- Pediatric Oncology
- Transplantation Medicine
Background:
- Sinusoidal obstructive syndrome (SOS) is a serious complication following hematopoietic cell transplantation (HCT).
- International consensus has established new diagnostic criteria and severity grading for SOS in pediatric and adolescent young adult (AYA) patients.
- Adoption and impact of these new criteria within the United States remain largely uncharacterized.
Purpose of the Study:
- To retrospectively assess the impact of newly endorsed international diagnostic criteria for SOS.
- To compare the incidence and timing of SOS diagnosis using historic criteria versus the new pEBMT guidelines in a US pediatric HCT cohort.
Main Methods:
- Single-center retrospective study of pediatric-AYA patients undergoing HCT between July 2009 and 2019.
- Application of historic Baltimore and Seattle diagnostic criteria for SOS.
- Comparison with the Paediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation (pEBMT) criteria.
Main Results:
- The pEBMT criteria identified a higher incidence of SOS (15.9%) compared to modified Seattle (12.3%) and Baltimore (6.6%) criteria.
- pEBMT criteria led to an earlier diagnosis of SOS by 2.5-3 days.
- 75% of patients had refractory thrombocytopenia at diagnosis; 61% were anicteric initially, but 94.4% developed hyperbilirubinemia.
- Application of pEBMT criteria could have prompted definitive treatment 3 days earlier.
Conclusions:
- The pEBMT diagnostic criteria are sensitive and highly specific for identifying SOS in pediatric-AYA HCT patients.
- These criteria may facilitate earlier diagnosis and treatment initiation, potentially improving outcomes.
- Further prospective studies are warranted to fully characterize SOS using the pEBMT criteria.
Abstract:
New diagnostic criteria and severity grading for sinusoidal obstructive syndrome (SOS) among pediatric and adolescent young adult (AYA) patients have been recently endorsed by international consensus. The extent to which these have been adopted in the US remains unclear. We sought to assess the potential impact via retrospective application of these criteria among patients treated at a large academic center in the United States. This is a single center retrospective study of pediatric-AYA patients who underwent hematopoietic cell transplantation (HCT) between July 2009 and 2019. The incidence of SOS was assessed using historic Baltimore and Seattle diagnostic criteria and compared with more recent guidelines (pEBMT) as proposed by the Paediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation. Among 226 patients, application of the pEBMT diagnostic criteria was associated with a higher incidence (15.9%) and earlier time to diagnosis of SOS (by 2.5-3 days) compared with the modified Seattle (12.3%), and Baltimore (6.6%) criteria, respectively. The pEBMT criteria were sensitive and highly specific. Refractory thrombocytopenia was present in 75% of patients at diagnosis. Approximately 61% of patients with SOS were anicteric at diagnosis, though the majority (94.4%) developed hyperbilirubinemia as SOS progressed over a median time of 4 (1-57) days. Application of pEBMT criteria may have resulted in earlier indication for definitive treatment by 3 days. Timely diagnosis and administration of definitive treatment of SOS has been associated with improved outcomes. Prospective studies may better characterize the risk factors and natural course of SOS using pEBMT criteria.
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