Eculizumab in secondary atypical haemolytic uraemic syndrome
Background. Complement dysregulation occurs in thrombotic microangiopathies (TMAs) other than primary atypical haemolytic uraemic syndrome (aHUS). A few of these patients have been reported previously to be successfully treated with eculizumab. Methods. We identified 29 patients with so-called secon...
| Autores: | , , , , , , , , , , , , , , , , , , , , , , , , , , , |
|---|---|
| Tipo de recurso: | artículo |
| Fecha de publicación: | 2017 |
| País: | España |
| Institución: | Universidad Autónoma de Madrid |
| Repositorio: | Biblos-e Archivo. Repositorio Institucional de la UAM |
| Idioma: | inglés |
| OAI Identifier: | oai:repositorio.uam.es:10486/680548 |
| Acceso en línea: | http://hdl.handle.net/10486/680548 https://dx.doi.org/10.1093/ndt/gfw453 |
| Access Level: | acceso abierto |
| Palabra clave: | Atypical haemolytic uraemic syndrome Complement activation Eculizumab Thrombotic microangiopathies Medicina |
| id |
ES_44b2ff688ebd9dd5cdcf350336b04c2e |
|---|---|
| oai_identifier_str |
oai:repositorio.uam.es:10486/680548 |
| network_acronym_str |
ES |
| network_name_str |
España |
| repository_id_str |
|
| spelling |
Eculizumab in secondary atypical haemolytic uraemic syndromeCavero, TeresaRabasco, CristinaLópez, AntíaRomán, ElenaÁvila, AnaSevillano, ÁngelHuerta, AnaRojas-Rivera, JorgeFuentes, CarolinaBlasco, MiquelJarque, AnaGarcía, AlbaMendizabal, SantiagoGavela, EvaMacía, ManuelQuintana, Luis F.Romera, Ana MaríaBorrego, JosefaArjona, EmiEspinosa, MarioPortolés Pérez, José MaríaGracia Iguacel, Carolina MartaGonzález Parra, Emilio JoséAljama, PedroMorales, EnriqueCao, MercedesRodríguez de Córdoba, SantiagoPraga, ManuelAtypical haemolytic uraemic syndromeComplement activationEculizumabThrombotic microangiopathiesMedicinaBackground. Complement dysregulation occurs in thrombotic microangiopathies (TMAs) other than primary atypical haemolytic uraemic syndrome (aHUS). A few of these patients have been reported previously to be successfully treated with eculizumab. Methods. We identified 29 patients with so-called secondary aHUS who had received eculizumab at 11 Spanish nephrology centres. Primary outcome was TMA resolution, defined by a normalization of platelet count (>150 × 109/L) and haemoglobin, disappearance of all the markers of microangiopathic haemolytic anaemia (MAHA), and improvement of renal function, with a ≥25% reduction of serum creatinine from the onset of eculizumab administration. Results. Twenty-nine patients with secondary aHUS (15 drug-induced, 8 associated with systemic diseases, 2 with postpartum, 2 with cancer-related, 1 associated with acute humoral rejection and 1 with intestinal lymphangiectasia) were included in this study. The reason to initiate eculizumab treatment was worsening of renal function and persistence of TMA despite treatment of the TMA cause and plasmapheresis. All patients showed severe MAHA and renal function impairment (14 requiring dialysis) prior to eculizumab treatment and 11 presented severe extrarenal manifestations. A rapid resolution of the TMA was observed in 20 patients (68%), 15 of them showing a ≥50% serum creatinine reduction at the last follow-up. Comprehensive genetic and molecular studies in 22 patients identified complement pathogenic variants in only 2 patients. With these two exceptions, eculizumab was discontinued, after a median of 8 weeks of treatment, without the occurrence of aHUS relapses. Conclusion. Short treatment with eculizumab can result in a rapid improvement of patients with secondary aHUS in whom TMA has persisted and renal function worsened despite treatment of the TMA-inducing conditionWork in this report was funded by the Instituto de Salud Carlos III: REDinREN (RD 016/009 Feder Funds), the Fondo de Investigaciones Sanitarias (13/02502 and ICI14/00350), the Ministerio de Economia y Competitividad (SAF2015-66287R) and the Autonomous Region of Madrid (S2010/BMD-2316; Grupo de Investigación Complemento-CM). SRdeC is funded by the Seventh Framework Programme European Union Project EURenOmics (305608)Oxford University Press on behalf of ERA-EDTA.Departamento de MedicinaFacultad de MedicinaInstituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD)20172017-02-20research articlehttp://purl.org/coar/resource_type/c_2df8fbb1VoRhttp://purl.org/coar/version/c_970fb48d4fbd8a85info:eu-repo/semantics/articleapplication/pdfhttp://hdl.handle.net/10486/680548https://dx.doi.org/10.1093/ndt/gfw453reponame:Biblos-e Archivo. Repositorio Institucional de la UAMinstname:Universidad Autónoma de MadridInglésengopen accesshttp://purl.org/coar/access_right/c_abf2info:eu-repo/semantics/openAccessoai:repositorio.uam.es:10486/6805482026-06-23T12:46:27Z |
| dc.title.none.fl_str_mv |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| title |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| spellingShingle |
Eculizumab in secondary atypical haemolytic uraemic syndrome Cavero, Teresa Atypical haemolytic uraemic syndrome Complement activation Eculizumab Thrombotic microangiopathies Medicina |
| title_short |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| title_full |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| title_fullStr |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| title_full_unstemmed |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| title_sort |
Eculizumab in secondary atypical haemolytic uraemic syndrome |
| dc.creator.none.fl_str_mv |
Cavero, Teresa Rabasco, Cristina López, Antía Román, Elena Ávila, Ana Sevillano, Ángel Huerta, Ana Rojas-Rivera, Jorge Fuentes, Carolina Blasco, Miquel Jarque, Ana García, Alba Mendizabal, Santiago Gavela, Eva Macía, Manuel Quintana, Luis F. Romera, Ana María Borrego, Josefa Arjona, Emi Espinosa, Mario Portolés Pérez, José María Gracia Iguacel, Carolina Marta González Parra, Emilio José Aljama, Pedro Morales, Enrique Cao, Mercedes Rodríguez de Córdoba, Santiago Praga, Manuel |
| author |
Cavero, Teresa |
| author_facet |
Cavero, Teresa Rabasco, Cristina López, Antía Román, Elena Ávila, Ana Sevillano, Ángel Huerta, Ana Rojas-Rivera, Jorge Fuentes, Carolina Blasco, Miquel Jarque, Ana García, Alba Mendizabal, Santiago Gavela, Eva Macía, Manuel Quintana, Luis F. Romera, Ana María Borrego, Josefa Arjona, Emi Espinosa, Mario Portolés Pérez, José María Gracia Iguacel, Carolina Marta González Parra, Emilio José Aljama, Pedro Morales, Enrique Cao, Mercedes Rodríguez de Córdoba, Santiago Praga, Manuel |
| author_role |
author |
| author2 |
Rabasco, Cristina López, Antía Román, Elena Ávila, Ana Sevillano, Ángel Huerta, Ana Rojas-Rivera, Jorge Fuentes, Carolina Blasco, Miquel Jarque, Ana García, Alba Mendizabal, Santiago Gavela, Eva Macía, Manuel Quintana, Luis F. Romera, Ana María Borrego, Josefa Arjona, Emi Espinosa, Mario Portolés Pérez, José María Gracia Iguacel, Carolina Marta González Parra, Emilio José Aljama, Pedro Morales, Enrique Cao, Mercedes Rodríguez de Córdoba, Santiago Praga, Manuel |
| author2_role |
author author author author author author author author author author author author author author author author author author author author author author author author author author author |
| dc.contributor.none.fl_str_mv |
Departamento de Medicina Facultad de Medicina Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD) |
| dc.subject.none.fl_str_mv |
Atypical haemolytic uraemic syndrome Complement activation Eculizumab Thrombotic microangiopathies Medicina |
| topic |
Atypical haemolytic uraemic syndrome Complement activation Eculizumab Thrombotic microangiopathies Medicina |
| description |
Background. Complement dysregulation occurs in thrombotic microangiopathies (TMAs) other than primary atypical haemolytic uraemic syndrome (aHUS). A few of these patients have been reported previously to be successfully treated with eculizumab. Methods. We identified 29 patients with so-called secondary aHUS who had received eculizumab at 11 Spanish nephrology centres. Primary outcome was TMA resolution, defined by a normalization of platelet count (>150 × 109/L) and haemoglobin, disappearance of all the markers of microangiopathic haemolytic anaemia (MAHA), and improvement of renal function, with a ≥25% reduction of serum creatinine from the onset of eculizumab administration. Results. Twenty-nine patients with secondary aHUS (15 drug-induced, 8 associated with systemic diseases, 2 with postpartum, 2 with cancer-related, 1 associated with acute humoral rejection and 1 with intestinal lymphangiectasia) were included in this study. The reason to initiate eculizumab treatment was worsening of renal function and persistence of TMA despite treatment of the TMA cause and plasmapheresis. All patients showed severe MAHA and renal function impairment (14 requiring dialysis) prior to eculizumab treatment and 11 presented severe extrarenal manifestations. A rapid resolution of the TMA was observed in 20 patients (68%), 15 of them showing a ≥50% serum creatinine reduction at the last follow-up. Comprehensive genetic and molecular studies in 22 patients identified complement pathogenic variants in only 2 patients. With these two exceptions, eculizumab was discontinued, after a median of 8 weeks of treatment, without the occurrence of aHUS relapses. Conclusion. Short treatment with eculizumab can result in a rapid improvement of patients with secondary aHUS in whom TMA has persisted and renal function worsened despite treatment of the TMA-inducing condition |
| publishDate |
2017 |
| dc.date.none.fl_str_mv |
2017 2017-02-20 |
| dc.type.none.fl_str_mv |
research article http://purl.org/coar/resource_type/c_2df8fbb1 VoR http://purl.org/coar/version/c_970fb48d4fbd8a85 |
| dc.type.openaire.fl_str_mv |
info:eu-repo/semantics/article |
| format |
article |
| dc.identifier.none.fl_str_mv |
http://hdl.handle.net/10486/680548 https://dx.doi.org/10.1093/ndt/gfw453 |
| url |
http://hdl.handle.net/10486/680548 https://dx.doi.org/10.1093/ndt/gfw453 |
| dc.language.none.fl_str_mv |
Inglés eng |
| language_invalid_str_mv |
Inglés |
| language |
eng |
| dc.rights.none.fl_str_mv |
open access http://purl.org/coar/access_right/c_abf2 |
| dc.rights.openaire.fl_str_mv |
info:eu-repo/semantics/openAccess |
| rights_invalid_str_mv |
open access http://purl.org/coar/access_right/c_abf2 |
| eu_rights_str_mv |
openAccess |
| dc.format.none.fl_str_mv |
application/pdf |
| dc.publisher.none.fl_str_mv |
Oxford University Press on behalf of ERA-EDTA. |
| publisher.none.fl_str_mv |
Oxford University Press on behalf of ERA-EDTA. |
| dc.source.none.fl_str_mv |
reponame:Biblos-e Archivo. Repositorio Institucional de la UAM instname:Universidad Autónoma de Madrid |
| instname_str |
Universidad Autónoma de Madrid |
| reponame_str |
Biblos-e Archivo. Repositorio Institucional de la UAM |
| collection |
Biblos-e Archivo. Repositorio Institucional de la UAM |
| repository.name.fl_str_mv |
|
| repository.mail.fl_str_mv |
|
| _version_ |
1869407111607222272 |
| score |
15.300719 |