Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial

Marisol Betensky; Maua Mosha; Cristina Tarango; Anupam Verma; Rukhmi Bhat; Nicole E. Kucine; Taizo Nakano; Charles Nakar; Gary Woods; Ernest Amankwah; Leonardo R. Brandão; Sam Schulman; Neil A. Goldenberg

doi:10.1182/bloodadvances.2024014415

Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial

Marisol Betensky^*, Maua Mosha, Cristina Tarango, Anupam Verma, Rukhmi Bhat, Nicole E. Kucine, Taizo Nakano, Charles Nakar, Gary Woods, Ernest Amankwah, Leonardo R. Brandão, Sam Schulman, Neil A. Goldenberg

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › peer-review

Abstract

Few studies have prospectively evaluated the incidence and outcomes in children with provoked venous thromboembolism (VTE) and transient or persistent antiphospholipid antibodies (aPLs). We compared outcomes of patients aged <21 years with a first-episode acute provoked VTE and positive aPL at diagnosis, enrolled in the Multicenter Evaluation of the Duration of Therapy for Thrombosis in Children trial. aPLs were tested at enrollment and, when positive, repeated at 6 weeks after VTE diagnosis. Subsequent testing was performed at the discretion of the treating hematologist. Of 524 patients, 116 (22%) had positive aPLs at enrollment. At follow-up, 70 (60%) had transient (n = 66) or low-titer aPLs (n = 4), 11 (10%) had persistent aPLs meeting the criteria for antiphospholipid antibody syndrome (APS), and 35 (30%) had no repeat testing. Patients with APS were older (15.8 vs 9.9 years; P = .014) and had a statistically significant higher risk of symptomatic recurrent VTE (18% vs 1%; odds ratio [OR], 12.2; 95% confidence interval [CI], 1.4-108; P = .025) and a statistically nonsignificant but clinically meaningful difference in the risk of anticoagulant-related clinically relevant bleeding (9% vs 0%; OR, 20.1; 95% CI, 0.7-558; P = .077) compared with those in the transient or low-titer aPL group. In conclusion, aPLs are common in young patients with acute provoked VTE and are mostly transitory and clinically insignificant. Patients with APS and provoked VTE appear to have an increased risk of recurrent VTE compared with patients with transitory or low-titer aPLs. Future collaborative studies should investigate the optimal VTE management for children with provoked VTE who meet the criteria for APS. The trial was registered at www.ClinicalTrials.gov as #NCT00687882.

Original language	English (US)
Pages (from-to)	5790-5795
Number of pages	6
Journal	Blood Advances
Volume	8
Issue number	22
DOIs	https://doi.org/10.1182/bloodadvances.2024014415
State	Published - Nov 26 2024

Funding

The authors thank the patients and families for their generous participation in the Kids-DOTT multicenter trial. The authors thank the members of the Kids-DOTT Executive Steering Committee and the Kids-DOTT Trial Investigators. The Kids-DOTT trial was supported by a U01 grant (1U01HL130048; Clinical Coordinating Center and Data Coordinating Center) from the National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI); and received support from an American Society of Hematology Bridge Grant, an Institutional Research Grant from the Johns Hopkins All Children\u2019s Foundation, an NIH NHLBI K23 award (1K23HL084055), and a Hemophilia and Thrombosis Research Society Thrombosis Studies Award. The Kids-DOTT trial was supported by a U01 grant (1U01HL130048; Clinical Coordinating Center and Data Coordinating Center) from the National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI); and received support from an American Society of Hematology Bridge Grant, an Institutional Research Grant from the Johns Hopkins All Children\u2019s Foundation, an NIH NHLBI K23 award (1K23HL084055), and a Hemophilia and Thrombosis Research Society Thrombosis Studies Award.

ASJC Scopus subject areas

Hematology

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10.1182/bloodadvances.2024014415

Cite this

Betensky, M., Mosha, M., Tarango, C., Verma, A., Bhat, R., Kucine, N. E., Nakano, T., Nakar, C., Woods, G., Amankwah, E., Brandão, L. R., Schulman, S., & Goldenberg, N. A. (2024). Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial. Blood Advances, 8(22), 5790-5795. https://doi.org/10.1182/bloodadvances.2024014415

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title = "Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial",

abstract = "Few studies have prospectively evaluated the incidence and outcomes in children with provoked venous thromboembolism (VTE) and transient or persistent antiphospholipid antibodies (aPLs). We compared outcomes of patients aged <21 years with a first-episode acute provoked VTE and positive aPL at diagnosis, enrolled in the Multicenter Evaluation of the Duration of Therapy for Thrombosis in Children trial. aPLs were tested at enrollment and, when positive, repeated at 6 weeks after VTE diagnosis. Subsequent testing was performed at the discretion of the treating hematologist. Of 524 patients, 116 (22%) had positive aPLs at enrollment. At follow-up, 70 (60%) had transient (n = 66) or low-titer aPLs (n = 4), 11 (10%) had persistent aPLs meeting the criteria for antiphospholipid antibody syndrome (APS), and 35 (30%) had no repeat testing. Patients with APS were older (15.8 vs 9.9 years; P = .014) and had a statistically significant higher risk of symptomatic recurrent VTE (18% vs 1%; odds ratio [OR], 12.2; 95% confidence interval [CI], 1.4-108; P = .025) and a statistically nonsignificant but clinically meaningful difference in the risk of anticoagulant-related clinically relevant bleeding (9% vs 0%; OR, 20.1; 95% CI, 0.7-558; P = .077) compared with those in the transient or low-titer aPL group. In conclusion, aPLs are common in young patients with acute provoked VTE and are mostly transitory and clinically insignificant. Patients with APS and provoked VTE appear to have an increased risk of recurrent VTE compared with patients with transitory or low-titer aPLs. Future collaborative studies should investigate the optimal VTE management for children with provoked VTE who meet the criteria for APS. The trial was registered at www.ClinicalTrials.gov as #NCT00687882.",

author = "Marisol Betensky and Maua Mosha and Cristina Tarango and Anupam Verma and Rukhmi Bhat and Kucine, {Nicole E.} and Taizo Nakano and Charles Nakar and Gary Woods and Ernest Amankwah and Brand{\~a}o, {Leonardo R.} and Sam Schulman and Goldenberg, {Neil A.}",

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Betensky, M, Mosha, M, Tarango, C, Verma, A, Bhat, R, Kucine, NE, Nakano, T, Nakar, C, Woods, G, Amankwah, E, Brandão, LR, Schulman, S & Goldenberg, NA 2024, 'Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial', Blood Advances, vol. 8, no. 22, pp. 5790-5795. https://doi.org/10.1182/bloodadvances.2024014415

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T1 - Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies

T2 - findings from the Kids-DOTT trial

AU - Betensky, Marisol

AU - Mosha, Maua

AU - Tarango, Cristina

AU - Verma, Anupam

AU - Bhat, Rukhmi

AU - Kucine, Nicole E.

AU - Nakano, Taizo

AU - Nakar, Charles

AU - Woods, Gary

AU - Amankwah, Ernest

AU - Brandão, Leonardo R.

AU - Schulman, Sam

AU - Goldenberg, Neil A.

PY - 2024/11/26

Y1 - 2024/11/26

N2 - Few studies have prospectively evaluated the incidence and outcomes in children with provoked venous thromboembolism (VTE) and transient or persistent antiphospholipid antibodies (aPLs). We compared outcomes of patients aged <21 years with a first-episode acute provoked VTE and positive aPL at diagnosis, enrolled in the Multicenter Evaluation of the Duration of Therapy for Thrombosis in Children trial. aPLs were tested at enrollment and, when positive, repeated at 6 weeks after VTE diagnosis. Subsequent testing was performed at the discretion of the treating hematologist. Of 524 patients, 116 (22%) had positive aPLs at enrollment. At follow-up, 70 (60%) had transient (n = 66) or low-titer aPLs (n = 4), 11 (10%) had persistent aPLs meeting the criteria for antiphospholipid antibody syndrome (APS), and 35 (30%) had no repeat testing. Patients with APS were older (15.8 vs 9.9 years; P = .014) and had a statistically significant higher risk of symptomatic recurrent VTE (18% vs 1%; odds ratio [OR], 12.2; 95% confidence interval [CI], 1.4-108; P = .025) and a statistically nonsignificant but clinically meaningful difference in the risk of anticoagulant-related clinically relevant bleeding (9% vs 0%; OR, 20.1; 95% CI, 0.7-558; P = .077) compared with those in the transient or low-titer aPL group. In conclusion, aPLs are common in young patients with acute provoked VTE and are mostly transitory and clinically insignificant. Patients with APS and provoked VTE appear to have an increased risk of recurrent VTE compared with patients with transitory or low-titer aPLs. Future collaborative studies should investigate the optimal VTE management for children with provoked VTE who meet the criteria for APS. The trial was registered at www.ClinicalTrials.gov as #NCT00687882.

AB - Few studies have prospectively evaluated the incidence and outcomes in children with provoked venous thromboembolism (VTE) and transient or persistent antiphospholipid antibodies (aPLs). We compared outcomes of patients aged <21 years with a first-episode acute provoked VTE and positive aPL at diagnosis, enrolled in the Multicenter Evaluation of the Duration of Therapy for Thrombosis in Children trial. aPLs were tested at enrollment and, when positive, repeated at 6 weeks after VTE diagnosis. Subsequent testing was performed at the discretion of the treating hematologist. Of 524 patients, 116 (22%) had positive aPLs at enrollment. At follow-up, 70 (60%) had transient (n = 66) or low-titer aPLs (n = 4), 11 (10%) had persistent aPLs meeting the criteria for antiphospholipid antibody syndrome (APS), and 35 (30%) had no repeat testing. Patients with APS were older (15.8 vs 9.9 years; P = .014) and had a statistically significant higher risk of symptomatic recurrent VTE (18% vs 1%; odds ratio [OR], 12.2; 95% confidence interval [CI], 1.4-108; P = .025) and a statistically nonsignificant but clinically meaningful difference in the risk of anticoagulant-related clinically relevant bleeding (9% vs 0%; OR, 20.1; 95% CI, 0.7-558; P = .077) compared with those in the transient or low-titer aPL group. In conclusion, aPLs are common in young patients with acute provoked VTE and are mostly transitory and clinically insignificant. Patients with APS and provoked VTE appear to have an increased risk of recurrent VTE compared with patients with transitory or low-titer aPLs. Future collaborative studies should investigate the optimal VTE management for children with provoked VTE who meet the criteria for APS. The trial was registered at www.ClinicalTrials.gov as #NCT00687882.

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Outcomes in children with provoked venous thrombosis and antiphospholipid antibodies: findings from the Kids-DOTT trial

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