Diagnosis, grading, and treatment recommendations for children, adolescents, and young adults with sinusoidal obstructive syndrome: an international expert position statement

Mahadeo, Kris Michael; Bajwa, Rajinder; Abdel‐Azim, Hisham; Lehmann, Leslie; Duncan, Christine; Zantek, Nicole D.; Vittorio, Jennifer; Angelo, Joseph; McArthur, Jennifer; Schadler, Keri; Chan, Sherwin S; Tewari, Priti; Khazal, Sajad; Auletta, Jeffery J.; Choi, Sung Won; Shoberu, Basirat; Kałwak, Krzysztof; Harden, Avis; Kebriaei, Partow; Abe, Jun‐ichi; Li, Shulin; Moffet, Jerelyn; Abraham, Susan C.; Tambaro, Francesco Paolo; Kleinschmidt, Katharina; Richardson, Paul G.; Corbacioglu, Selim; Transplantation, Marrow

doi:10.1016/s2352-3026(19)30201-7

Cited by 65 publications

(69 citation statements)

References 67 publications

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“…The tool has been validated for up to 21 years of age and developmental anchor points may be used to ensure appropriate assessments (14). Recently, CAPD has been suggested for potential use in adolescent & young adult patients up to 25 years of age (15). CAPD is specifically used in lieu of the ICE score for ICANS assessment in children (13).…”

Section: Introductionmentioning

confidence: 99%

Immune Effector Cell Associated Neurotoxicity (ICANS) in Pediatric and Young Adult Patients Following Chimeric Antigen Receptor (CAR) T-Cell Therapy: Can We Optimize Early Diagnosis?

et al. 2021

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The Cornell Assessment for Pediatric Delirium (CAPD) was first proposed by the Pediatric Acute Lung Injury and Sepsis Investigators Network-Stem Cell Transplantation and Cancer Immunotherapy Subgroup and MD Anderson CARTOX joint working committees, for detection of immune effector cell associated neurotoxicity (ICANS) in pediatric patients receiving chimeric antigen receptor (CAR) T-cell therapy. It was subsequently adopted by the American Society for Transplantation and Cellular Therapy. The utility of CAPD as a screening tool for early diagnosis of ICANS has not been fully characterized. We conducted a retrospective study of pediatric and young adult patients (n=15) receiving standard-of-care CAR T-cell products. Cytokine release syndrome (CRS) and ICANS occurred in 87% and 40% of patients, respectively. ICANS was associated with significantly higher peaks of serum ferritin. A change in CAPD from a prior baseline was noted in 60% of patients with ICANS, 24–72 h prior to diagnosis of ICANS. The median change from baseline to maximum CAPD score of patients who developed ICANS versus those who did not was 13 versus 3, respectively (p=0.0004). Changes in CAPD score from baseline may be the earliest indicator of ICANS among pediatric and young adult patients which may warrant closer monitoring, with more frequent CAPD assessments.

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Section: Introductionmentioning

confidence: 99%

Immune Effector Cell Associated Neurotoxicity (ICANS) in Pediatric and Young Adult Patients Following Chimeric Antigen Receptor (CAR) T-Cell Therapy: Can We Optimize Early Diagnosis?

et al. 2021

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“…Doppler-mode characteristics include portal vein flow demodulation, decreased flow velocity or reversal of portal vein flow, decreased spectral density, increased resistive index and peak systolic velocity of the hepatic artery, congestion index, monophasic reduced flow in the hepatic veins, and visualization of collateral veins (e.g., in the paraumbilical veins). These characteristics are summarized in Table 2 [1,5,[15][16][17][18][19][20]. For the Doppler examination, a right intercostal approach is preferred because the Doppler angles for performing velocity measurements and evaluation of flow are optimal.…”

Section: Ultrasonographymentioning

confidence: 99%

“…Reduced, monophasic flow in right hepatic vein [17] measured >8 cm from inferior vena cava [19] Ascites Minimal, moderate or need for external drainage [1] Mild: minimal fluid by liver, spleen or pelvis Moderate: <1 cm fluid. Severe: fluid in all three regions, >1 cm in ≥2 regions [5] Visualization of collateral veins E.g., in the paraumbilical veins, seen as a hypoechoic lumen in the hyperechoic round ligament [2,15,17,19] a Higher risk group: busulfan conditioning regimen before hematopoietic stem cell transplantation (HSCT) b Lower risk group: no busulfan conditioning regimen before HSCT SD standard deviation flow reversal may precede clinical manifestations [25]. Later, flow velocity can decrease, show a to-and-fro pattern or ultimately reverse completely (Fig.…”

Section: Hepatic Vein Flowmentioning

confidence: 99%

Imaging features of hepatic sinusoidal obstruction syndrome or veno-occlusive disease in children

et al. 2021

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Hepatic sinusoidal obstruction syndrome, also known as veno-occlusive disease, can occur as a complication of myeloablative chemotherapy, as a result of low-intensity chemotherapy-related liver toxicity or radiotherapy of the liver. Symptoms of sinusoidal obstruction syndrome can range from asymptomatic to liver dysfunction or severe disease with life-threatening acute multi-organ failure. Imaging features can suggest or support this clinical diagnosis. Familiarity with the imaging spectrum of sinusoidal obstruction syndrome is therefore important for both radiologists and clinical oncologists. Here, multi-modality radiologic appearances of sinusoidal obstruction syndrome in pediatric patients are illustrated, including outcome after follow-up.

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“…Sinusoidal obstruction syndrome (SOS) of the liver is a serious, potentially life-threatening complication occurring usually within the first 30 days after hematopoietic stem cell transplantation (HSCT) [ 1 ] or after treatment with some antineoplastic agents without HSCT [ 2 ]. Diagnostic criteria are based on clinical and laboratory findings including weight gain/ascites, hyperbilirubinemia, and hepatomegaly/right upper abdominal quadrant pain (Seattle criteria [ 3 ] and Baltimore criteria [ 4 ]).…”

Section: Introductionmentioning

confidence: 99%

“…Defibrotide has been successfully used as prophylaxis in patients deemed to be at an increased risk for SOS due to pre-existing liver disease, an underlying condition, or treatment factors [ 9 ]. Prophylactic ursodeoxycholic acid has shown efficacy in the reduction in SOS and mortality [ 1 ].…”

Section: Introductionmentioning

confidence: 99%

Genetic Predictors for Sinusoidal Obstruction Syndrome—A Systematic Review

Waespe

Strebel

Mlakar

et al. 2021

JPM

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Sinusoidal obstruction syndrome (SOS) is a potentially life-threatening complication after hematopoietic stem cell transplantation (HSCT) or antineoplastic treatment without HSCT. Genetic variants were investigated for their association with SOS, but the evidence is inconclusive. We performed a systematic literature review to identify genes, gene variants, and methods of association analyses of genetic markers with SOS. We identified 23 studies after HSCT and 4 studies after antineoplastic treatment without HSCT. One study (4%) performed whole-exome sequencing (WES) and replicated the analysis in an independent cohort, 26 used a candidate-gene approach. Three studies included >200 participants (11%), and six were of high quality (22%). Variants in 34 genes were tested in candidate gene studies after HSCT. Variants in GSTA1 were associated with SOS in three studies, MTHFR in two, and CPS1, CTH, CYP2B6, GSTM1, GSTP1, HFE, and HPSE in one study each. UGT2B10 and LNPK variants were identified in a WES analysis. After exposure to antineoplastic agents without HSCT, variants in six genes were tested and only GSTM1 was associated with SOS. There was a substantial heterogeneity of populations within and between studies. Future research should be based on sufficiently large homogenous samples, adjust for covariates, and replicate findings in independent cohorts.

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Diagnosis, grading, and treatment recommendations for children, adolescents, and young adults with sinusoidal obstructive syndrome: an international expert position statement

Cited by 65 publications

References 67 publications

Immune Effector Cell Associated Neurotoxicity (ICANS) in Pediatric and Young Adult Patients Following Chimeric Antigen Receptor (CAR) T-Cell Therapy: Can We Optimize Early Diagnosis?

Immune Effector Cell Associated Neurotoxicity (ICANS) in Pediatric and Young Adult Patients Following Chimeric Antigen Receptor (CAR) T-Cell Therapy: Can We Optimize Early Diagnosis?

Imaging features of hepatic sinusoidal obstruction syndrome or veno-occlusive disease in children

Genetic Predictors for Sinusoidal Obstruction Syndrome—A Systematic Review

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