A Retrospective, Observational Study on the Response to Caplacizumab Treatment in aTTP Patients: the Italian Experience (ROSCAPLI) : A Retrospective, Observational Study on the Response to Caplacizumab Treatment in aTTP Patients: the Italian Experience (ROSCAPLI)

This study is a multicenter, retrospective, observational study in patients with acquired thrombotic thrombocytopenic purpura (aTTP) treated with plasma exchange (PEX) in association with caplacizumab, and immunosuppression between Q4-2019 and 28 February2021.Study designThe retrospective study will measure the following vital, clinical chemistry, hematological, and hemostatic parameters, according to the consensus guidelines of aTTP diagnosis and as a part of routine diagnostic work-up:Age, sex, BMI, blood pressure at diagnosisPlatelet count at diagnosis and at the follow up visitsHb level at diagnosis at the follow up visitsWhite blood cell count at diagnosis at the follow up visitsCreatinine at diagnosis at the follow up visitsschistocytes count at diagnosis at the follow up visitsLDH at diagnosis at the follow up visitsCoombs' assay at diagnosisalanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visitstotal bilirubin at diagnosis at the follow up visitsTroponin above ULN at any pointADAMTS13 activity (where measured) at diagnosis at the follow up visitsAnti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits The clinical signs will be parametrized by the PLASMIC score and the Glasgow score, where calculated in the various centers. The PLASMIC score and the Glasgow score will be evaluated according to the obtained values (0-4, 5-6, and 7; <13 and 13-15, respectively). All the clinical and laboratory parameters will be taken in each center and reported in the CRF.OBJECTIVES The primary objective in this study is the description and quantification of clinical response in terms of platelet count recovery in patients with aTTP treated t with caplacizumab , in addition to PEX and immunosuppression in the real-world setting. The Secondary objectives include: Number of exacerbations, defined as recurrent thrombocytopenia within 30 days after the end of therapy; Rate of relapse, defined as a TTP event occurring more than 30 days after the end of daily plasma exchange;Refractoriness; defined by the lack of a doubling of platelet count after 4 days of treatment and a lactate dehydrogenase level that remained above the upper limit of the normal range, TTP-related mortality and evaluation of adverse events.Data collection and management A customized eCRF (RedCap) will be created for the study and only the principal investigator and collaborating investigators of the center will have access to patient medical records during the study.STATISTICAL CONSIDERATIONS Qualitative variables will be expressed by absolute and percentage frequency. Quantitative variables will be previously assessed for their distribution by the Shapiro-Wilk test. Normally distributed data will be described by mean and standard deviation (SD), whilst non-normally distributed variable by median and interquartile range (IQR).INFORMED CONSENT Informed consent form will be collected for all except for all patients whom death is ascertained.

Medienart:	Klinische Studie

Erscheinungsjahr:	2022
Erschienen:	2022

Enthalten in:	ClinicalTrials.gov - (2022) vom: 02. März Zur Gesamtaufnahme - year:2022

Sprache:	Englisch

Links:	Volltext [kostenfrei]

Themen:	610 Medical Condition: Thrombotic Thrombocytopenic Purpura, Acquired Purpura Purpura, Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Recruitment Status: Not yet recruiting Study Type: Observational

Anmerkungen:	Source: Link to the current ClinicalTrials.gov record., First posted: March 2, 2022, Last downloaded: ClinicalTrials.gov processed this data on March 14, 2022, Last updated: March 16, 2022

Study ID:

NCT05262881 4305

Veröffentlichungen zur Studie:

Scully M, Hunt BJ, Benjamin S, Liesner R, Rose P, Peyvandi F, Cheung B, Machin SJ; British Committee for Standards in Haematology. Guidelines on the diagnosis and management of thrombotic thrombocytopenic purpura and other thrombotic microangiopathies. Br J Haematol. 2012 Aug;158(3):323-35. doi: 10.1111/j.1365-2141.2012.09167.x. Epub 2012 May 25. No abstract available. Rizzo C, Rizzo S, Scire E, Di Bona D, Ingrassia C, Franco G, Bono R, Quintini G, Caruso C. Thrombotic thrombocytopenic purpura: a review of the literature in the light of our experience with plasma exchange. Blood Transfus. 2012 Oct;10(4):521-32. doi: 10.2450/2012.0122-11. Epub 2012 Jun 27. No abstract available. Bell WR, Braine HG, Ness PM, Kickler TS. Improved survival in thrombotic thrombocytopenic purpura-hemolytic uremic syndrome. Clinical experience in 108 patients. N Engl J Med. 1991 Aug 8;325(6):398-403. doi: 10.1056/NEJM199108083250605. Nguyen L, Li X, Duvall D, Terrell DR, Vesely SK, George JN. Twice-daily plasma exchange for patients with refractory thrombotic thrombocytopenic purpura: the experience of the Oklahoma Registry, 1989 through 2006. Transfusion. 2008 Feb;48(2):349-57. doi: 10.1111/j.1537-2995.2007.01530.x. Epub 2007 Nov 19. Balduini CL, Gugliotta L, Luppi M, Laurenti L, Klersy C, Pieresca C, Quintini G, Iuliano F, Re R, Spedini P, Vianelli N, Zaccaria A, Pogliani EM, Musso R, Bobbio Pallavicini E, Quarta G, Galieni P, Fragasso A, Casella G, Noris P, Ascari E; Italian TTP Study Group. High versus standard dose methylprednisolone in the acute phase of idiopathic thrombotic thrombocytopenic purpura: a randomized study. Ann Hematol. 2010 Jun;89(6):591-6. doi: 10.1007/s00277-009-0877-5. Epub 2009 Dec 23. 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 Jan;45(1):41-9. doi: 10.1111/j.1537-2995.2005.03146.x. Beloncle F, Buffet M, Coindre JP, Munoz-Bongrand N, Malot S, Pene F, Mira JP, Galicier L, Guidet B, Baudel JL, Subra JF, Tanguy-Schmidt A, Pourrat J, Azoulay E, Veyradier A, Coppo P; Thrombotic Microangiopathies Reference Center. Splenectomy and/or cyclophosphamide as salvage therapies in thrombotic thrombocytopenic purpura: the French TMA Reference Center experience. Transfusion. 2012 Nov;52(11):2436-44. doi: 10.1111/j.1537-2995.2012.03578.x. Epub 2012 Mar 8. Sayani FA, Abrams CS. How I treat refractory thrombotic thrombocytopenic purpura. Blood. 2015 Jun 18;125(25):3860-7. doi: 10.1182/blood-2014-11-551580. Epub 2015 Mar 17. Erratum In: Blood. 2017 Oct 5;130(14 ):1684. Lim W, Vesely SK, George JN. The role of rituximab in the management of patients with acquired thrombotic thrombocytopenic purpura. Blood. 2015 Mar 5;125(10):1526-31. doi: 10.1182/blood-2014-10-559211. Epub 2015 Jan 8. No abstract available. Vazquez-Mellado A, Pequeno-Luevano M, Cantu-Rodriguez OG, Villarreal-Martinez L, Jaime-Perez JC, Gomez-De-Leon A, De La Garza-Salazar F, Gonzalez-Llano O, Colunga-Pedraza P, Sotomayor-Duque G, Gomez-Almaguer D. More about low-dose rituximab and plasma exchange as front-line therapy for patients with thrombotic thrombocytopenic purpura. Hematology. 2016 Jun;21(5):311-6. doi: 10.1080/10245332.2015.1133008. Epub 2016 Feb 24. Scully M, McDonald V, Cavenagh J, Hunt BJ, Longair I, Cohen H, Machin SJ. A phase 2 study of the safety and efficacy of rituximab with plasma exchange in acute acquired thrombotic thrombocytopenic purpura. Blood. 2011 Aug 18;118(7):1746-53. doi: 10.1182/blood-2011-03-341131. Epub 2011 Jun 2. Page EE, Kremer Hovinga JA, Terrell DR, Vesely SK, George JN. Rituximab reduces risk for relapse in patients with thrombotic thrombocytopenic purpura. Blood. 2016 Jun 16;127(24):3092-4. doi: 10.1182/blood-2016-03-703827. Epub 2016 Apr 8. No abstract available. Hie M, Gay J, Galicier L, Provot F, Presne C, Poullin P, Bonmarchand G, Wynckel A, Benhamou Y, Vanhille P, Servais A, Bordessoule D, Coindre JP, Hamidou M, Vernant JP, Veyradier A, Coppo P; French Thrombotic Microangiopathies Reference Centre. Preemptive rituximab infusions after remission efficiently prevent relapses in acquired thrombotic thrombocytopenic purpura. Blood. 2014 Jul 10;124(2):204-10. doi: 10.1182/blood-2014-01-550244. Epub 2014 May 28. Rottenstreich A, Hochberg-Klein S, Rund D, Kalish Y. The role of N-acetylcysteine in the treatment of thrombotic thrombocytopenic purpura. J Thromb Thrombolysis. 2016 May;41(4):678-83. doi: 10.1007/s11239-015-1259-6. Eskazan AE. Bortezomib therapy in patients with relapsed/refractory acquired thrombotic thrombocytopenic purpura. Ann Hematol. 2016 Oct;95(11):1751-6. doi: 10.1007/s00277-016-2804-x. Epub 2016 Sep 3. Masias C, Cataland SR. Novel therapies in thrombotic thrombocytopenic purpura. Res Pract Thromb Haemost. 2017 Dec 18;2(1):19-26. doi: 10.1002/rth2.12066. eCollection 2018 Jan. Volker LA, Kaufeld J, Miesbach W, Brahler S, Reinhardt M, Kuhne L, Muhlfeld A, Schreiber A, Gaedeke J, Tolle M, Jabs WJ, Ozcan F, Markau S, Girndt M, Bauer F, Westhoff TH, Felten H, Hausberg M, Brand M, Gerth J, Bieringer M, Bommer M, Zschiedrich S, Schneider J, Elitok S, Gawlik A, Gackler A, Kribben A, Schwenger V, Schoenermarck U, Roeder M, Radermacher J, Bramstedt J, Morgner A, Herbst R, Harth A, Potthoff SA, von Auer C, Wendt R, Christ H, Brinkkoetter PT, Menne J. Real-world data confirm the effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura. Blood Adv. 2020 Jul 14;4(13):3085-3092. doi: 10.1182/bloodadvances.2020001973. Erratum In: Blood Adv. 2022 Apr 12;6(7):2434. Jamme M, Rondeau E. The PLASMIC score for thrombotic thrombocytopenic purpura. Lancet Haematol. 2017 Apr;4(4):e148-e149. doi: 10.1016/S2352-3026(17)30024-8. Epub 2017 Mar 2. No abstract available.

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PPN (Katalog-ID):	CTG008253471