Authors' recommendations: The use of eculizumab in PNH patients when there is no clinical evidence should be considered as experimental. These situations include at this moment: patients without a history of transfusions, patients under 18, pregnant patients, and cases of Eculizumab for treatment of PNH patients who recommence eculizumab treatment after withdrawal. Given that there is only solid evidence of eculizumab efficacy in surrogated endpoints, transfused PRBC units, and LDH levels, these outcomes should guide the criteria that define those patients who would qualify for eculizumab treatment. There is solid evidence that only patients with a greater history of transfusions in the previous year and higher LDH baseline levels, show major clinical benefit and better relation cost-efficacy. In consequence, those should be the criteria to select subgroups which potentially will obtain more benefit from treatment. For the rest of patients supportive treatment and monitoring are recommended. In areas where there is uncertainty about eculizumab efficacy, being it measured in terms of health-related outcomes (f. e. efficacy in rates of thrombosis), it is recommended to establish an agreement based on shared risks with the manufacturing laboratory. It is necessary for all patients who enroll for eculizumab treatment to accomplish the Risk Management Plan designed by the EMA for this drug. It is recommended to undertake an evaluation of efficacy and safety after 3 months of treatment. In this initial evaluation, LDH levels will indicate eculizumab efficacy. In those patients in which eculizumab is not effective and/or safe, it is recommended to withdraw treatment. Patients in whom treatment seems to be effective after the three first months should continue periodic monitorisation every six months. The following parameters should be registered: thrombotic events (type and location), number of RBC units transfused, biochemical parameters (LDH and creatinine), hemograma, infections (type and location), disease evolving towards other haematologic pathologies (aplastic anemia, leukaemia or myelodysplasic syndromes), adverse events, and spontaneus remission. Patients for whom eculizumab is not effective and/or safe are recommended to withdraw from treatment. It is recommendable that the SSPA creates a group for the granting of centralised authorisation for this treatment. The group should apply the resolution established by the SAS management, individually, to each potential case. In the same way this group will be responsible for management of patients' monitoring. In addition, it is recommended the foundation of a PNH registry in Andalusia.

Project Status: Completed

URL for project: https://www.aetsa.org/publicacion/eculizumab-soliris-evaluacion-de-la-eficacia-y-seguridad-del-medicamento-y-analisis-economico-de-su-uso-para-el-tratamiento-de-la-hemoglobinuria-paroxistica-nocturna/

Year Published: 2011

URL for published report: https://www.aetsa.org/download/publicaciones/antiguas/AETSA_5-2011_Eculizumab_01.pdf

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: Spain

Organisation Name: Andalusian Health Technology Assessment Area

Contact Address: Area de Evaluacion de Tecnologias Sanitarias Sanitarias de Andalucia (AETSA) Avda. Innovación, s/n Edificio Arena 1. Sevilla (Spain) Tel. +34 955 006 309

Contact Name: aetsa.csalud@juntadeandalucia.es

Contact Email: aetsa.csalud@juntadeandalucia.es

Copyright: Andalusian Agency for Health Technology Assessment (AETSA)