Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2)

Peter Collins; Francesco Baudo; Paul Knoebl; Herve Levesque; Laszlo Nemes; Fabio Pellegrini; Angela Huth-Kuehne; Gerold Aspoeck; Max Heistinger; Paul Knobl; Anne Makipernaa; Helene Andre; A Aouba; Sylvia Bellucci; Philippe Beurrier; Jeanne Yvonne Borg; Luc Darnige; Jean Devignes; Roseline dOiron; Philippe Gautier; Valerie Gay; Stephane Girault; Yves Gruel; Viviane Guerin; Nathalie Hezard; Mehdi Khellaf; Martial Koenig; Francois Lifermann; Raphael Marlu; J. Ninet; Jocelyne Peynet; Thomas Quemeneur; Chantal Rothschild; Nicolas Schleinitz; Marianne Sigaud; Sebastien Trouillier; Sophie Voisin; Andreas Giebl; Katharina Holstein; Angela Huth-Kuhne; Ralph M. Loreth; Udo Steigerwald; Andreas Tiede; George Theodossiades; Gaspar Radvanyi; Agota Schlammadinger; Giovanni Barillari; Samantha Pasca; T. Caimi; L. Contino; Armando D'Angelo; Luciano Crippa; Annalisa Fattorini; Giovanni Di Minno; Anna Maria Cerbone; Matteo Nicola Dario Di Minno; Marco D'inca; Anna Falanga; Anna Maggioni; Teresa Lerede; Massimo Franchini; Gianluca Gaidano; Lorenzo De Paoli; Gabriella Gamba; Raffaele Ghirardi; Mauro Girotto; Delios Tasca; Elvira Grandone; Giovanni Tiscia; Davide Imberti; Alfonso Iorio; Raffaele Landolfi; Leonardo Di Gennaro; Linda Novarese; Guglielmo Mariani; Mario Lapecorella; Marco Marietta; Paola Pedrazzi; Maria Gabriella Mazzucconi; Cristina Santoro; Massimo Morfini; Silvia Linari; Stefano Moratelli; Rossella Paolini; Gavino Piseddu; Renzo Poggio; Enrico Pogliani; Monica Carpenedo; Chiara Remiddi; Elena Santagostino; Maria Elisa Mancuso; Rita Santoro; Giuseppina Papaleo; Piercarla Schinco; Alessandra Borchiellini; Federica Valeri; Anna Rita Scortechini; Sergio Siragusa; Gianluca Sottilotta; Alessandro Squizzato; Giuseppe Tagariello; Roberto Sartori; Anna Rita Tagliaferri; Caterina Di Perna; Gianna Franca Rivolta; Sophie Testa; Oriana Paoletti; Vincenzo Toschi; Ezio Zanon; Barbara Brandolin; Karly Hamulyak; Pieter Kamphuisen; Britta Laros-van Gorkom; Frank W.G. Leebeek; Nijziel Marten; Irena Novakova; Roger Schutgens; P.W.G van der Linden; Joost van Esser; J. van der Meer; Paula Ypma; Manuel Campos; Carlos Aguilar; Carmen Altisent; Nuria Bermejo; Raquel Del Campo; M. Ferreiro Arguelles; Rosario Gonzalez Boullosa; Maria Jose Gutierrez Pimentel; Victor Jimenez-Yuste; Lucia Jose-Felix; Pascual Marco; Maria Eva Mingot; Rosario Perez Garrido; Noelia z Perez Gonzale; Manuel Prieto Garcia; Ana Maria Rodriguez-Huerta; HGUG Maranon; Carmen Sedano; Alexandra Tolosa Munoz; Fariba Baghaei; Lilian Tengborn; Francoise Boehlen; Wolfgang Korte; Pratima Chowdary; Gillian Evans; Suzanne Pavord; Savita Rangarajan; Jonathan Wilde

doi:10.1182/blood-2012-02-409185

Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2)

Peter Collins^*, Francesco Baudo, Paul Knoebl, Herve Levesque, Laszlo Nemes, Fabio Pellegrini, Angela Huth-Kuehne, Gerold Aspoeck, Max Heistinger, Paul Knobl, Anne Makipernaa, Helene Andre, A Aouba, Sylvia Bellucci, Philippe Beurrier, Jeanne Yvonne Borg, Luc Darnige, Jean Devignes, Roseline dOiron, Philippe GautierValerie Gay, Stephane Girault, Yves Gruel, Viviane Guerin, Nathalie Hezard, Mehdi Khellaf, Martial Koenig, Francois Lifermann, Raphael Marlu, J. Ninet, Jocelyne Peynet, Thomas Quemeneur, Chantal Rothschild, Nicolas Schleinitz, Marianne Sigaud, Sebastien Trouillier, Sophie Voisin, Andreas Giebl, Katharina Holstein, Angela Huth-Kuhne, Ralph M. Loreth, Udo Steigerwald, Andreas Tiede, George Theodossiades, Gaspar Radvanyi, Agota Schlammadinger, Giovanni Barillari, Samantha Pasca, T. Caimi, L. Contino, Armando D'Angelo, Luciano Crippa, Annalisa Fattorini, Giovanni Di Minno, Anna Maria Cerbone, Matteo Nicola Dario Di Minno, Marco D'inca, Anna Falanga, Anna Maggioni, Teresa Lerede, Massimo Franchini, Gianluca Gaidano, Lorenzo De Paoli, Gabriella Gamba, Raffaele Ghirardi, Mauro Girotto, Delios Tasca, Elvira Grandone, Giovanni Tiscia, Davide Imberti, Alfonso Iorio, Raffaele Landolfi, Leonardo Di Gennaro, Linda Novarese, Guglielmo Mariani, Mario Lapecorella, Marco Marietta, Paola Pedrazzi, Maria Gabriella Mazzucconi, Cristina Santoro, Massimo Morfini, Silvia Linari, Stefano Moratelli, Rossella Paolini, Gavino Piseddu, Renzo Poggio, Enrico Pogliani, Monica Carpenedo, Chiara Remiddi, Elena Santagostino, Maria Elisa Mancuso, Rita Santoro, Giuseppina Papaleo, Piercarla Schinco, Alessandra Borchiellini, Federica Valeri, Anna Rita Scortechini, Sergio Siragusa, Gianluca Sottilotta, Alessandro Squizzato, Giuseppe Tagariello, Roberto Sartori, Anna Rita Tagliaferri, Caterina Di Perna, Gianna Franca Rivolta, Sophie Testa, Oriana Paoletti, Vincenzo Toschi, Ezio Zanon, Barbara Brandolin, Karly Hamulyak, Pieter Kamphuisen, Britta Laros-van Gorkom, Frank W.G. Leebeek, Nijziel Marten, Irena Novakova, Roger Schutgens, P.W.G van der Linden, Joost van Esser, J. van der Meer, Paula Ypma, Manuel Campos, Carlos Aguilar, Carmen Altisent, Nuria Bermejo, Raquel Del Campo, M. Ferreiro Arguelles, Rosario Gonzalez Boullosa, Maria Jose Gutierrez Pimentel, Victor Jimenez-Yuste, Lucia Jose-Felix, Pascual Marco, Maria Eva Mingot, Rosario Perez Garrido, Noelia z Perez Gonzale, Manuel Prieto Garcia, Ana Maria Rodriguez-Huerta, HGUG Maranon, Carmen Sedano, Alexandra Tolosa Munoz, Fariba Baghaei, Lilian Tengborn, Francoise Boehlen, Wolfgang Korte, Pratima Chowdary, Gillian Evans, Suzanne Pavord, Savita Rangarajan, Jonathan Wilde

^*Corresponding author for this work

Research output: Contribution to journal › Article › Academic › peer-review

182 Citations (Web of Science)

Abstract

Acquired hemophilia A (AHA) is an autoimmune disease caused by an autoantibody to factor VIII. Patients are at risk of severe and fatal hemorrhage until the inhibitor is eradicated, and guidelines recommend immunosuppression as soon as the diagnosis has been made. The optimal immunosuppressive regimen is unclear; therefore, data from 331 patients entered into the prospective EACH2 registry were analyzed. Steroids combined with cyclophosphamide resulted in more stable complete remission (70%), defined as inhibitor undetectable, factor VIII more than 70 IU/dL and immunosuppression stopped, than steroids alone (48%) or rituximab-based regimens (59%). Propensity score-matched analysis controlling for age, sex, factor VIII level, inhibitor titer, and underlying etiology confirmed that stable remission was more likely with steroids and cyclophosphamide than steroids alone (odds ratio = 3.25; 95% CI, 1.51-6.96; P <.003). The median time to complete remission was approximately 5 weeks for steroids with or without cyclo-phosphamide; rituximab-based regimens required approximately twice as long. Immunoglobulin administration did not improve outcome. Second-line therapy was successful in approximately 60% of cases that failed first- line therapy. Outcome was not affected by the choice of first-line therapy. The likelihood of achieving stable remission was not affected by underlying etiology but was influenced by the presenting inhibitor titer and FVIII level. (Blood. 2012;120(1):47-55)

Original language	English
Pages (from-to)	47-55
Journal	Blood
Volume	120
Issue number	1
DOIs	https://doi.org/10.1182/blood-2012-02-409185
Publication status	Published - 5 Jul 2012

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10.1182/blood-2012-02-409185

Cite this

Collins, P., Baudo, F., Knoebl, P., Levesque, H., Nemes, L., Pellegrini, F., Huth-Kuehne, A., Aspoeck, G., Heistinger, M., Knobl, P., Makipernaa, A., Andre, H., Aouba, A., Bellucci, S., Beurrier, P., Borg, J. Y., Darnige, L., Devignes, J., dOiron, R., ... Wilde, J. (2012). Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2). Blood, 120(1), 47-55. https://doi.org/10.1182/blood-2012-02-409185

@article{0f703f03436146f3afd73141c093aff9,

title = "Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2)",

abstract = "Acquired hemophilia A (AHA) is an autoimmune disease caused by an autoantibody to factor VIII. Patients are at risk of severe and fatal hemorrhage until the inhibitor is eradicated, and guidelines recommend immunosuppression as soon as the diagnosis has been made. The optimal immunosuppressive regimen is unclear; therefore, data from 331 patients entered into the prospective EACH2 registry were analyzed. Steroids combined with cyclophosphamide resulted in more stable complete remission (70%), defined as inhibitor undetectable, factor VIII more than 70 IU/dL and immunosuppression stopped, than steroids alone (48%) or rituximab-based regimens (59%). Propensity score-matched analysis controlling for age, sex, factor VIII level, inhibitor titer, and underlying etiology confirmed that stable remission was more likely with steroids and cyclophosphamide than steroids alone (odds ratio = 3.25; 95% CI, 1.51-6.96; P <.003). The median time to complete remission was approximately 5 weeks for steroids with or without cyclo-phosphamide; rituximab-based regimens required approximately twice as long. Immunoglobulin administration did not improve outcome. Second-line therapy was successful in approximately 60% of cases that failed first- line therapy. Outcome was not affected by the choice of first-line therapy. The likelihood of achieving stable remission was not affected by underlying etiology but was influenced by the presenting inhibitor titer and FVIII level. (Blood. 2012;120(1):47-55)",

author = "Peter Collins and Francesco Baudo and Paul Knoebl and Herve Levesque and Laszlo Nemes and Fabio Pellegrini and Angela Huth-Kuehne and Gerold Aspoeck and Max Heistinger and Paul Knobl and Anne Makipernaa and Helene Andre and A Aouba and Sylvia Bellucci and Philippe Beurrier and Borg, {Jeanne Yvonne} and Luc Darnige and Jean Devignes and Roseline dOiron and Philippe Gautier and Valerie Gay and Stephane Girault and Yves Gruel and Viviane Guerin and Nathalie Hezard and Mehdi Khellaf and Martial Koenig and Francois Lifermann and Raphael Marlu and J. Ninet and Jocelyne Peynet and Thomas Quemeneur and Chantal Rothschild and Nicolas Schleinitz and Marianne Sigaud and Sebastien Trouillier and Sophie Voisin and Andreas Giebl and Katharina Holstein and Angela Huth-Kuhne and Loreth, {Ralph M.} and Udo Steigerwald and Andreas Tiede and George Theodossiades and Gaspar Radvanyi and Agota Schlammadinger and Giovanni Barillari and Samantha Pasca and T. Caimi and L. Contino and Armando D'Angelo and Luciano Crippa and Annalisa Fattorini and {Di Minno}, Giovanni and Cerbone, {Anna Maria} and {Di Minno}, {Matteo Nicola Dario} and Marco D'inca and Anna Falanga and Anna Maggioni and Teresa Lerede and Massimo Franchini and Gianluca Gaidano and {De Paoli}, Lorenzo and Gabriella Gamba and Raffaele Ghirardi and Mauro Girotto and Delios Tasca and Elvira Grandone and Giovanni Tiscia and Davide Imberti and Alfonso Iorio and Raffaele Landolfi and {Di Gennaro}, Leonardo and Linda Novarese and Guglielmo Mariani and Mario Lapecorella and Marco Marietta and Paola Pedrazzi and Mazzucconi, {Maria Gabriella} and Cristina Santoro and Massimo Morfini and Silvia Linari and Stefano Moratelli and Rossella Paolini and Gavino Piseddu and Renzo Poggio and Enrico Pogliani and Monica Carpenedo and Chiara Remiddi and Elena Santagostino and Mancuso, {Maria Elisa} and Rita Santoro and Giuseppina Papaleo and Piercarla Schinco and Alessandra Borchiellini and Federica Valeri and Scortechini, {Anna Rita} and Sergio Siragusa and Gianluca Sottilotta and Alessandro Squizzato and Giuseppe Tagariello and Roberto Sartori and Tagliaferri, {Anna Rita} and {Di Perna}, Caterina and Rivolta, {Gianna Franca} and Sophie Testa and Oriana Paoletti and Vincenzo Toschi and Ezio Zanon and Barbara Brandolin and Karly Hamulyak and Pieter Kamphuisen and {Laros-van Gorkom}, Britta and Leebeek, {Frank W.G.} and Nijziel Marten and Irena Novakova and Roger Schutgens and {van der Linden}, P.W.G and {van Esser}, Joost and {van der Meer}, J. and Paula Ypma and Manuel Campos and Carlos Aguilar and Carmen Altisent and Nuria Bermejo and {Del Campo}, Raquel and {Ferreiro Arguelles}, M. and {Gonzalez Boullosa}, Rosario and {Gutierrez Pimentel}, {Maria Jose} and Victor Jimenez-Yuste and Lucia Jose-Felix and Pascual Marco and Mingot, {Maria Eva} and {Perez Garrido}, Rosario and {Perez Gonzale}, {Noelia z} and {Prieto Garcia}, Manuel and Rodriguez-Huerta, {Ana Maria} and HGUG Maranon and Carmen Sedano and {Tolosa Munoz}, Alexandra and Fariba Baghaei and Lilian Tengborn and Francoise Boehlen and Wolfgang Korte and Pratima Chowdary and Gillian Evans and Suzanne Pavord and Savita Rangarajan and Jonathan Wilde",

year = "2012",

month = jul,

day = "5",

doi = "10.1182/blood-2012-02-409185",

language = "English",

volume = "120",

pages = "47--55",

journal = "Blood",

issn = "0006-4971",

publisher = "The American Society of Hematology",

number = "1",

}

Collins, P, Baudo, F, Knoebl, P, Levesque, H, Nemes, L, Pellegrini, F, Huth-Kuehne, A, Aspoeck, G, Heistinger, M, Knobl, P, Makipernaa, A, Andre, H, Aouba, A, Bellucci, S, Beurrier, P, Borg, JY, Darnige, L, Devignes, J, dOiron, R, Gautier, P, Gay, V, Girault, S, Gruel, Y, Guerin, V, Hezard, N, Khellaf, M, Koenig, M, Lifermann, F, Marlu, R, Ninet, J, Peynet, J, Quemeneur, T, Rothschild, C, Schleinitz, N, Sigaud, M, Trouillier, S, Voisin, S, Giebl, A, Holstein, K, Huth-Kuhne, A, Loreth, RM, Steigerwald, U, Tiede, A, Theodossiades, G, Radvanyi, G, Schlammadinger, A, Barillari, G, Pasca, S, Caimi, T, Contino, L, D'Angelo, A, Crippa, L, Fattorini, A, Di Minno, G, Cerbone, AM, Di Minno, MND, D'inca, M, Falanga, A, Maggioni, A, Lerede, T, Franchini, M, Gaidano, G, De Paoli, L, Gamba, G, Ghirardi, R, Girotto, M, Tasca, D, Grandone, E, Tiscia, G, Imberti, D, Iorio, A, Landolfi, R, Di Gennaro, L, Novarese, L, Mariani, G, Lapecorella, M, Marietta, M, Pedrazzi, P, Mazzucconi, MG, Santoro, C, Morfini, M, Linari, S, Moratelli, S, Paolini, R, Piseddu, G, Poggio, R, Pogliani, E, Carpenedo, M, Remiddi, C, Santagostino, E, Mancuso, ME, Santoro, R, Papaleo, G, Schinco, P, Borchiellini, A, Valeri, F, Scortechini, AR, Siragusa, S, Sottilotta, G, Squizzato, A, Tagariello, G, Sartori, R, Tagliaferri, AR, Di Perna, C, Rivolta, GF, Testa, S, Paoletti, O, Toschi, V, Zanon, E, Brandolin, B, Hamulyak, K, Kamphuisen, P, Laros-van Gorkom, B, Leebeek, FWG, Marten, N, Novakova, I, Schutgens, R, van der Linden, PWG, van Esser, J, van der Meer, J, Ypma, P, Campos, M, Aguilar, C, Altisent, C, Bermejo, N, Del Campo, R, Ferreiro Arguelles, M, Gonzalez Boullosa, R, Gutierrez Pimentel, MJ, Jimenez-Yuste, V, Jose-Felix, L, Marco, P, Mingot, ME, Perez Garrido, R, Perez Gonzale, NZ, Prieto Garcia, M, Rodriguez-Huerta, AM, Maranon, HGUG, Sedano, C, Tolosa Munoz, A, Baghaei, F, Tengborn, L, Boehlen, F, Korte, W, Chowdary, P, Evans, G, Pavord, S, Rangarajan, S & Wilde, J 2012, 'Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2)', Blood, vol. 120, no. 1, pp. 47-55. https://doi.org/10.1182/blood-2012-02-409185

TY - JOUR

T1 - Immunosuppression for acquired hemophilia A: results from the European Acquired Haemophilia Registry (EACH2)

AU - Collins, Peter

AU - Baudo, Francesco

AU - Knoebl, Paul

AU - Levesque, Herve

AU - Nemes, Laszlo

AU - Pellegrini, Fabio

AU - Huth-Kuehne, Angela

AU - Aspoeck, Gerold

AU - Heistinger, Max

AU - Knobl, Paul

AU - Makipernaa, Anne

AU - Andre, Helene

AU - Aouba, A

AU - Bellucci, Sylvia

AU - Beurrier, Philippe

AU - Borg, Jeanne Yvonne

AU - Darnige, Luc

AU - Devignes, Jean

AU - dOiron, Roseline

AU - Gautier, Philippe

AU - Gay, Valerie

AU - Girault, Stephane

AU - Gruel, Yves

AU - Guerin, Viviane

AU - Hezard, Nathalie

AU - Khellaf, Mehdi

AU - Koenig, Martial

AU - Lifermann, Francois

AU - Marlu, Raphael

AU - Ninet, J.

AU - Peynet, Jocelyne

AU - Quemeneur, Thomas

AU - Rothschild, Chantal

AU - Schleinitz, Nicolas

AU - Sigaud, Marianne

AU - Trouillier, Sebastien

AU - Voisin, Sophie

AU - Giebl, Andreas

AU - Holstein, Katharina

AU - Huth-Kuhne, Angela

AU - Loreth, Ralph M.

AU - Steigerwald, Udo

AU - Tiede, Andreas

AU - Theodossiades, George

AU - Radvanyi, Gaspar

AU - Schlammadinger, Agota

AU - Barillari, Giovanni

AU - Pasca, Samantha

AU - Caimi, T.

AU - Contino, L.

AU - D'Angelo, Armando

AU - Crippa, Luciano

AU - Fattorini, Annalisa

AU - Di Minno, Giovanni

AU - Cerbone, Anna Maria

AU - Di Minno, Matteo Nicola Dario

AU - D'inca, Marco

AU - Falanga, Anna

AU - Maggioni, Anna

AU - Lerede, Teresa

AU - Franchini, Massimo

AU - Gaidano, Gianluca

AU - De Paoli, Lorenzo

AU - Gamba, Gabriella

AU - Ghirardi, Raffaele

AU - Girotto, Mauro

AU - Tasca, Delios

AU - Grandone, Elvira

AU - Tiscia, Giovanni

AU - Imberti, Davide

AU - Iorio, Alfonso

AU - Landolfi, Raffaele

AU - Di Gennaro, Leonardo

AU - Novarese, Linda

AU - Mariani, Guglielmo

AU - Lapecorella, Mario

AU - Marietta, Marco

AU - Pedrazzi, Paola

AU - Mazzucconi, Maria Gabriella

AU - Santoro, Cristina

AU - Morfini, Massimo

AU - Linari, Silvia

AU - Moratelli, Stefano

AU - Paolini, Rossella

AU - Piseddu, Gavino

AU - Poggio, Renzo

AU - Pogliani, Enrico

AU - Carpenedo, Monica

AU - Remiddi, Chiara

AU - Santagostino, Elena

AU - Mancuso, Maria Elisa

AU - Santoro, Rita

AU - Papaleo, Giuseppina

AU - Schinco, Piercarla

AU - Borchiellini, Alessandra

AU - Valeri, Federica

AU - Scortechini, Anna Rita

AU - Siragusa, Sergio

AU - Sottilotta, Gianluca

AU - Squizzato, Alessandro

AU - Tagariello, Giuseppe

AU - Sartori, Roberto

AU - Tagliaferri, Anna Rita

AU - Di Perna, Caterina

AU - Rivolta, Gianna Franca

AU - Testa, Sophie

AU - Paoletti, Oriana

AU - Toschi, Vincenzo

AU - Zanon, Ezio

AU - Brandolin, Barbara

AU - Hamulyak, Karly

AU - Kamphuisen, Pieter

AU - Laros-van Gorkom, Britta

AU - Leebeek, Frank W.G.

AU - Marten, Nijziel

AU - Novakova, Irena

AU - Schutgens, Roger

AU - van der Linden, P.W.G

AU - van Esser, Joost

AU - van der Meer, J.

AU - Ypma, Paula

AU - Campos, Manuel

AU - Aguilar, Carlos

AU - Altisent, Carmen

AU - Bermejo, Nuria

AU - Del Campo, Raquel

AU - Ferreiro Arguelles, M.

AU - Gonzalez Boullosa, Rosario

AU - Gutierrez Pimentel, Maria Jose

AU - Jimenez-Yuste, Victor

AU - Jose-Felix, Lucia

AU - Marco, Pascual

AU - Mingot, Maria Eva

AU - Perez Garrido, Rosario

AU - Perez Gonzale, Noelia z

AU - Prieto Garcia, Manuel

AU - Rodriguez-Huerta, Ana Maria

AU - Maranon, HGUG

AU - Sedano, Carmen

AU - Tolosa Munoz, Alexandra

AU - Baghaei, Fariba

AU - Tengborn, Lilian

AU - Boehlen, Francoise

AU - Korte, Wolfgang

AU - Chowdary, Pratima

AU - Evans, Gillian

AU - Pavord, Suzanne

AU - Rangarajan, Savita

AU - Wilde, Jonathan

PY - 2012/7/5

Y1 - 2012/7/5

N2 - Acquired hemophilia A (AHA) is an autoimmune disease caused by an autoantibody to factor VIII. Patients are at risk of severe and fatal hemorrhage until the inhibitor is eradicated, and guidelines recommend immunosuppression as soon as the diagnosis has been made. The optimal immunosuppressive regimen is unclear; therefore, data from 331 patients entered into the prospective EACH2 registry were analyzed. Steroids combined with cyclophosphamide resulted in more stable complete remission (70%), defined as inhibitor undetectable, factor VIII more than 70 IU/dL and immunosuppression stopped, than steroids alone (48%) or rituximab-based regimens (59%). Propensity score-matched analysis controlling for age, sex, factor VIII level, inhibitor titer, and underlying etiology confirmed that stable remission was more likely with steroids and cyclophosphamide than steroids alone (odds ratio = 3.25; 95% CI, 1.51-6.96; P <.003). The median time to complete remission was approximately 5 weeks for steroids with or without cyclo-phosphamide; rituximab-based regimens required approximately twice as long. Immunoglobulin administration did not improve outcome. Second-line therapy was successful in approximately 60% of cases that failed first- line therapy. Outcome was not affected by the choice of first-line therapy. The likelihood of achieving stable remission was not affected by underlying etiology but was influenced by the presenting inhibitor titer and FVIII level. (Blood. 2012;120(1):47-55)

AB - Acquired hemophilia A (AHA) is an autoimmune disease caused by an autoantibody to factor VIII. Patients are at risk of severe and fatal hemorrhage until the inhibitor is eradicated, and guidelines recommend immunosuppression as soon as the diagnosis has been made. The optimal immunosuppressive regimen is unclear; therefore, data from 331 patients entered into the prospective EACH2 registry were analyzed. Steroids combined with cyclophosphamide resulted in more stable complete remission (70%), defined as inhibitor undetectable, factor VIII more than 70 IU/dL and immunosuppression stopped, than steroids alone (48%) or rituximab-based regimens (59%). Propensity score-matched analysis controlling for age, sex, factor VIII level, inhibitor titer, and underlying etiology confirmed that stable remission was more likely with steroids and cyclophosphamide than steroids alone (odds ratio = 3.25; 95% CI, 1.51-6.96; P <.003). The median time to complete remission was approximately 5 weeks for steroids with or without cyclo-phosphamide; rituximab-based regimens required approximately twice as long. Immunoglobulin administration did not improve outcome. Second-line therapy was successful in approximately 60% of cases that failed first- line therapy. Outcome was not affected by the choice of first-line therapy. The likelihood of achieving stable remission was not affected by underlying etiology but was influenced by the presenting inhibitor titer and FVIII level. (Blood. 2012;120(1):47-55)

U2 - 10.1182/blood-2012-02-409185

DO - 10.1182/blood-2012-02-409185

M3 - Article

C2 - 22517903

VL - 120

SP - 47

EP - 55

JO - Blood

JF - Blood

SN - 0006-4971

IS - 1

ER -