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

NCT05262881 | Unknown

• 1 other identifier: 4305
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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. The retrospective study will measure: Age, sex, BMI, blood pressure at diagnosis, Platelet count at diagnosis and at the follow up visits, Hb level at diagnosis at the follow up visits, White blood cell count at diagnosis at the follow up visits, Creatinine at diagnosis at the follow up visits, schistocytes count at diagnosis at the follow up visits, LDH at diagnosis at the follow up visits, Coombs' assay at diagnosis, alanine-leucine-amino-transferase (ALT) at diagnosis at the follow up visits, total bilirubin at diagnosis at the follow up visits, Troponin above ULN at any point, ADAMTS13 activity (where measured) at diagnosis at the follow up visits, Anti-ADAMTS13 antibodies (where measured) at diagnosis at the follow up visits. 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.

Conditions

Thrombotic Thrombocytopenic Purpura, Acquired

Outcome Measures

Primary Outcomes (1)

• Time-to-response of Treatment Defined by a Confirmed Recovery of Platelets

  Platelet response was defined as recovery of platelets ≥ 150,000/µL.

  18 months

Secondary Outcomes (1)

• study of critical parameters

  18 months

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population consists of patients treated for acute aTTP episodes in the hematology, internal medicine, and critical care centers between Q4-2019 and Q1-2021 in Italy.

You may qualify if:

• The patients included in this study should have received caplacizumab for treatment in the period between Q4-2019 and the end of February 2021 while the end of follow up observation is scheduled for Q1-2021 (to observe at least one month of post treatment follow up). The diagnosis should be based on either clinical/laboratory parameters inclusive of measurement of ADAMTS13 level \<10%) or the PLASMIC score (platelets, lysis, active cancer, stem cell or solid organ transplant, MCV, INR, and creatinine) with intermediate and high risk (sore\>5) already computed or retrospectively calculated as previously detailed for centers that did not measure the ADAMTS13 level.

Sponsors & Collaborators

• Catholic University of the Sacred Heart: lead

Study Sites (1)

Fondazione Policlinico Universitario Agostino Gemelli, IRCCS

Rome, 00168, Italy

Location

Related Publications (18)

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  PMID: 22624596 RESULT

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  PMID: 22790258 RESULT

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  PMID: 2062331 RESULT

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  PMID: 18028271 RESULT

• 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.

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  PMID: 15647017 RESULT

• 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.

  PMID: 22404639 RESULT

• 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.

  PMID: 25784681 RESULT

• 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.

  PMID: 25573992 RESULT

• 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.

  PMID: 26907228 RESULT

• 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.

  PMID: 21636861 RESULT

• 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.

  PMID: 27060171 RESULT

• 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.

  PMID: 24869941 RESULT

• 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.

  PMID: 26245827 RESULT

• 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.

  PMID: 27590601 RESULT

• 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.

  PMID: 30046703 RESULT

• 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.

  PMID: 32634236 RESULT

• 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.

  PMID: 28259521 RESULT

Related Links

• European-Medicines-Agency. Cablivi (caplacizumab): summary of product characteristics. Document of the European Medicines Agency. 2018;

MeSH Terms

Conditions

Thrombotic thrombocytopenic purpura, acquired

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 21, 2022
• First Posted: March 2, 2022
• Study Start: March 1, 2022
• Primary Completion: September 30, 2022
• Study Completion: October 30, 2022
• Last Updated: March 2, 2022

Record last verified: 2022-02

Locations

IT (1)