Acquired thrombotic thrombocytopenic purpura in a 16-year-old patient
use of proteasome inhibitor for refractory disease control
DOI:
https://doi.org/10.47456/rbps.v26i1.51121Keywords:
Thrombotic Thrombocytopenic Purpura, Therapeutics, Bortezomib, AdolescentAbstract
Introduction: Thrombotic thrombocytopenic purpura (TTP) is a rare and severe disease characterized by the formation of abnormal blood clots that can lead to damage in various organs. It is marked by reduced ADAMTS13 activity levels and can be either acquired or congenital. Although conventional treatment with immunosuppressants and plasmapheresis is effective in most cases, a subgroup of patients develops refractory forms. The scarcity of therapeutic options for these patients, especially in resource-limited settings, makes managing refractory TTP challenging. We describe the case of an adolescent with acquired refractory TTP who achieved a complete response to treatment with bortezomib, focusing on age, diagnosis, therapy for refractory TTP, and its association with Systemic Lupus Erythematosus. Objective: To share this case with healthcare professionals in order to increase knowledge about refractory TTP and propose the inclusion of bortezomib as an alternative therapeutic option in selected cases. Methods: This is a descriptive observational study approved by the Medical Ethics Committee, with data obtained from the medical records of the Cassiano Antônio Moraes University Hospital (HUCAM). Results: Complete response after bortezomib therapy and the diagnosis of Systemic Lupus Erythematosus after 12 months of outpatient follow-up. Conclusion: In patients with acquired refractory TTP, the use of bortezomib shows efficacy and an appropriate safety profile, and should be considered in this scenario, as it is available in most specialized healthcare services.
Downloads
References
Fakhouri F, Vernant JP, Veyradier A, Wolf M, Kaplanski G, Binaut R, et al. Efficiency of curative and prophylactic treatment with rituximab in ADAMTS13-deficient thrombotic thrombocytopenic purpura: a study of 11 cases. Blood. 2005;106(6):1932–7. doi:10.1182/blood-2005-03-0848
Joly BS, Coppo P, Veyradier A. Thrombotic thrombocytopenic purpura. Blood. 2017;129(21):2836–46. doi:10.1182/blood-2016-10-709857
Upadhyay VA, Geisler BP, Sun L, Uhl L, Kaufman RM, Stowell C, et al. Using a PLASMIC scoring-based approach in the management of suspected immune thrombotic thrombocytopenic purpura: a cost-minimization analysis within the Harvard TMA Research Collaborative. Br J Haematol. 2019;186(3):490–8. doi:10.1111/bjh.15932
Zheng XL, Vesely SK, Cataland SR, Coppo P, Geldziler B, Iorio A, et al. ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura. J Thromb Haemost. 2020;18(10):2486–95. doi:10.1111/jth.15006
Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura. Blood. 2015;125(25):3860–7. doi:10.1182/blood-2014-11-551580
Chen H, Fu A, Wang J, Wu T, Li Z, Tang J, et al. Rituximab as first-line treatment for acquired thrombotic thrombocytopenic purpura. J Int Med Res. 2017;45(3):1253–60. doi:10.1177/0300060517695646
Zwicker JI, Muia J, Dolatshahi L, Westfield LA, Nieters P, Rodrigues A, et al. Adjuvant low-dose rituximab and plasma exchange for acquired TTP. Blood. 2019;134(13):1106–9. doi:10.1182/blood.2019000795
van Balen T, Schreuder MF, de Jong H, van de Kar NC. Refractory thrombotic thrombocytopenic purpura in a 16-year-old girl: successful treatment with bortezomib. Eur J Haematol. 2014;92(1):80–2. doi:10.1111/ejh.12206
Ziman A, Mitri M, Klapper E, Pepkowitz SH, Goldfinger D. Combination vincristine and plasma exchange as initial therapy in patients with thrombotic thrombocytopenic purpura: one institution’s experience and review of the literature. Transfusion. 2005;45:41–9. doi:10.1111/j.1537-2995.2005.03146.x
Öngören S, Salihoğlu A, Apaydın T, Sadri S, Eşkazan AE, Ar MC, et al. Vincristine as an adjunct to therapeutic plasma exchange for thrombotic thrombocytopenic purpura: a single-institution experience. Balkan Med J. 2018;35:417–21. doi:10.4274/balkanmedj.2017.1215
Lee NCJ, Yates S, Rambally S, Sarode R, Ibrahim IF, Shen YM, et al. Bortezomib in relapsed/refractory autoimmune-mediated thrombotic thrombocytopenic purpura: a single-center case series and systematic review. Blood. 2022;140(1):2693–4. doi:10.1182/blood-2022-165329
Patriquin CJ, Thomas MR, Dutt T, McGuckin S, Blombery PA, Cranfield T, et al. Bortezomib in the treatment of refractory thrombotic thrombocytopenic purpura. Br J Haematol. 2016;173:779–85. doi:10.1111/bjh.13993
Downloads
Published
Issue
Section
License
Copyright (c) 2024 Brazilian Journal of Health Research
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.
The Revista Brasileira de Pesquisa em Saúde (RBPS) adopts the CC BY 4.0 license, which means that authors retain the copyright of the works they submit to the journal. Authors are responsible for declaring that their contribution is an original manuscript, that it has not been previously published, and that it is not under simultaneous review by another scientific journal. Upon submitting the manuscript, authors grant RBPS the exclusive right of first publication, subject to peer review.
Authors are permitted to enter into additional contracts for the non-exclusive distribution of the version published by RBPS (for example, in institutional repositories or as a book chapter), provided that due acknowledgment of authorship and of initial publication by RBPS is given. Authors are also encouraged to make their work available online (for example, in institutional repositories or on their personal pages) after its initial publication in the journal, with due acknowledgment of authorship and of the original publication by RBPS.
Accordingly, under the CC BY 4.0 license, readers have the right to:
- Share — copy and redistribute the material in any medium or format for any purpose, even commercially;
- Adapt — remix, transform, and build upon the material for any purpose, even commercially.
The licensor cannot revoke these freedoms as long as you follow the license terms. Under the following terms:
- Attribution — You must give appropriate credit, provide a link to the license, and indicate if changes were made. You may do so in any reasonable manner, but not in any way that suggests the licensor endorses you or your use.
- No additional restrictions — You may not apply legal terms or technological measures that legally restrict others from doing anything the license permits.
