Should we consider caplacizumab as routine treatment for acute thrombotic thrombocytopenic purpura? An expert perspective on the pros and cons

ABSTRACT

Introduction

Immune thrombotic thrombocytopenic purpura (iTTP) is a rare and life-threatening disorder. Caplacizumab has been the latest drug incorporated into the initial treatment of acute episodes, allowing for faster platelet recovery and a decrease in refractoriness, exacerbation, thromboembolic events, and mortality. However, caplacizumab is also associated with a bleeding risk and higher treatment costs, which prevent many centers from using it universally.

Areas covered

Studies that included iTTP and/or caplacizumab to date were selected for this review using PubMed and MEDLINE platforms. We describe outcomes in the pre-caplacizumab era and after it, highlighting the benefits and risks of its use early in frontline, and also pointing out special situations that require careful management.

Expert opinion

It is clear that the availability of caplacizumab has significantly and favorably impacted the management of iTTP patients. Whether this improvement is cost-effective still remains uncertain, and data on long-term sequelae and different healthcare systems will help to clarify this point. In addition, evidence of the bleeding/thrombotic risk of iTTP patients under this drug needs to be better addressed in future studies.

KEYWORDS:

• Caplacizumab
• thrombotic thrombocytopenic purpura
• iTTP
• therapy
• outcome
• ADAMTS13

Article highlights

• Caplacizumab is currently the only approved drug for the treatment of adult patients with iTTP.

• Administration of caplacizumab to the standard of care therapy in patients with an acute episode of iTTP is associated with a reduced risk of refractory disease, shortened time to response, and improved exacerbation rates.

• Caplacizumab appears to increase the relapse rate and bleeding risk, and there are some contradictory data about the cost-effectiveness of its use in every patient with an acute iTTP episode.

• Additional data from clinical trials and real-world evidence are needed to finally define the role of caplacizumab in patients with iTTP.

Declaration of interests

I Gómez Seguí has participated in an advisory board for Takeda and received a speaker bureau from Sanofi. J de la Rubia is a consultant for Bristol-Meyers Squibb, GlaxoSmithKline, Janssen, Menarini, Pfizer, Sanofi, and Takeda and is on the advisory board of GlaxoSmithKline, Janssen, Pfizer, and Sanofi. M Eva Mingot Castellano has received grant/research support from Sanofi, Novartis, Takeda, Amgen, and Novo Nordisk. M Eva Mingot Castellano has also received advisory board and speaker bureau from Novartis, Sanofi, Sobi, Takeda, Grifols, Amgen, Shiomi, and Novo Nordisk. C Pascual Izquierdo has received advisory board and speaker bureau from Novartis, Sanofi, Sobi, Takeda, and Amgen, and grant/research support from Sanofi. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

A reviewer on this manuscript has disclosed they have previously received consultancy fees/advisory boards at Sanofi. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Additional information

Funding

This paper was not funded.