Aplasie midollari acquisite in età pediatrica

Raccomandazioni diagnostico-terapeutiche

Translated title of the contribution: Acquired aplastic anaemia in children: Diagnostic-therapeutic recommendations

Angelica Barone, Annunziata Lucarelli, Daniela Onofrillo, Federico Verzegnassi, Sonia Bonanomi, Daniela Longoni, Simone Cesaro, Chiara Cugno, Marco Zecca, Francesca Fioredda, Johanna Svahn, Carlo Dufour, Anna Paola Iori, Saverio Ladogana, Matteo Maruzzi, Anna Locasciulli, Marina Lanciotti, Alessandra Macaluso, Rosalba Mandaglio, Nicoletta Marra & 11 others Giuseppe Menna, Baldo Martire, Lucia Dora Notarangelo, Giovanni Palazzi, Marta Pillon, Ugo Ramenghi, Paola Saracco, Giovanna Russo, Fabio Timeus, Fabio Tucci, Piero Farruggia

Research output: Contribution to journalArticle

Abstract

Acquired Aplastic Anaemia (AA) is a rare heterogeneous disease characterized by pancytopoenia and hypoplastic bone marrow. The incidence is 2-3 millions per year (all age groups) in Europe, but is higher in East Asia. The pathogenesis of AA is complex and involves haematopoietic stem cell/progenitor cell deficiencies and autoimmune mechanism. Survival in severe aplastic anaemia (SAA) has markedly improved in the past 2 decades because of advances in haematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA haematopoietic stem cell transplant (HSCT) from a matched sibling donor (MSD) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. The objective of this guideline is to provide healthcare professionals with clear guidance on the diagnosis and management of paediatric patients with AA. A preliminary, evidence-based document issued by a group of paediatric haematologists was discussed, modified and approved during a series of "Consensus Conferences" according to procedures previously validated by the AIEOP Board. The guidelines highlight the importance of referring paediatric patients with AA to paediatric centres with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA; haematopoietic stem cell transplantation as first line therapy if an MSD is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CsA) if an MSD is not identified.

Original languageItalian
Pages (from-to)383-389
Number of pages7
JournalMedico e Bambino
Volume33
Issue number6
Publication statusPublished - 2014
Externally publishedYes

Fingerprint

Aplastic Anemia
Siblings
Immunosuppressive Agents
Hematopoietic Stem Cells
Tissue Donors
Pediatrics
Hematopoietic Stem Cell Transplantation
Therapeutics
Guidelines
Transplants
Unrelated Donors
Aftercare
Antilymphocyte Serum
Far East
Rare Diseases
Cyclosporine
Horses
Consensus
Differential Diagnosis
Stem Cells

Keywords

  • Acquired aplastic anemia
  • Childhood

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

Cite this

Barone, A., Lucarelli, A., Onofrillo, D., Verzegnassi, F., Bonanomi, S., Longoni, D., ... Farruggia, P. (2014). Aplasie midollari acquisite in età pediatrica: Raccomandazioni diagnostico-terapeutiche. Medico e Bambino, 33(6), 383-389.

Aplasie midollari acquisite in età pediatrica : Raccomandazioni diagnostico-terapeutiche. / Barone, Angelica; Lucarelli, Annunziata; Onofrillo, Daniela; Verzegnassi, Federico; Bonanomi, Sonia; Longoni, Daniela; Cesaro, Simone; Cugno, Chiara; Zecca, Marco; Fioredda, Francesca; Svahn, Johanna; Dufour, Carlo; Iori, Anna Paola; Ladogana, Saverio; Maruzzi, Matteo; Locasciulli, Anna; Lanciotti, Marina; Macaluso, Alessandra; Mandaglio, Rosalba; Marra, Nicoletta; Menna, Giuseppe; Martire, Baldo; Notarangelo, Lucia Dora; Palazzi, Giovanni; Pillon, Marta; Ramenghi, Ugo; Saracco, Paola; Russo, Giovanna; Timeus, Fabio; Tucci, Fabio; Farruggia, Piero.

In: Medico e Bambino, Vol. 33, No. 6, 2014, p. 383-389.

Research output: Contribution to journalArticle

Barone, A, Lucarelli, A, Onofrillo, D, Verzegnassi, F, Bonanomi, S, Longoni, D, Cesaro, S, Cugno, C, Zecca, M, Fioredda, F, Svahn, J, Dufour, C, Iori, AP, Ladogana, S, Maruzzi, M, Locasciulli, A, Lanciotti, M, Macaluso, A, Mandaglio, R, Marra, N, Menna, G, Martire, B, Notarangelo, LD, Palazzi, G, Pillon, M, Ramenghi, U, Saracco, P, Russo, G, Timeus, F, Tucci, F & Farruggia, P 2014, 'Aplasie midollari acquisite in età pediatrica: Raccomandazioni diagnostico-terapeutiche', Medico e Bambino, vol. 33, no. 6, pp. 383-389.
Barone A, Lucarelli A, Onofrillo D, Verzegnassi F, Bonanomi S, Longoni D et al. Aplasie midollari acquisite in età pediatrica: Raccomandazioni diagnostico-terapeutiche. Medico e Bambino. 2014;33(6):383-389.
Barone, Angelica ; Lucarelli, Annunziata ; Onofrillo, Daniela ; Verzegnassi, Federico ; Bonanomi, Sonia ; Longoni, Daniela ; Cesaro, Simone ; Cugno, Chiara ; Zecca, Marco ; Fioredda, Francesca ; Svahn, Johanna ; Dufour, Carlo ; Iori, Anna Paola ; Ladogana, Saverio ; Maruzzi, Matteo ; Locasciulli, Anna ; Lanciotti, Marina ; Macaluso, Alessandra ; Mandaglio, Rosalba ; Marra, Nicoletta ; Menna, Giuseppe ; Martire, Baldo ; Notarangelo, Lucia Dora ; Palazzi, Giovanni ; Pillon, Marta ; Ramenghi, Ugo ; Saracco, Paola ; Russo, Giovanna ; Timeus, Fabio ; Tucci, Fabio ; Farruggia, Piero. / Aplasie midollari acquisite in età pediatrica : Raccomandazioni diagnostico-terapeutiche. In: Medico e Bambino. 2014 ; Vol. 33, No. 6. pp. 383-389.
@article{11ce8d4f0fba4b07844caf7023d9df53,
title = "Aplasie midollari acquisite in et{\`a} pediatrica: Raccomandazioni diagnostico-terapeutiche",
abstract = "Acquired Aplastic Anaemia (AA) is a rare heterogeneous disease characterized by pancytopoenia and hypoplastic bone marrow. The incidence is 2-3 millions per year (all age groups) in Europe, but is higher in East Asia. The pathogenesis of AA is complex and involves haematopoietic stem cell/progenitor cell deficiencies and autoimmune mechanism. Survival in severe aplastic anaemia (SAA) has markedly improved in the past 2 decades because of advances in haematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA haematopoietic stem cell transplant (HSCT) from a matched sibling donor (MSD) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. The objective of this guideline is to provide healthcare professionals with clear guidance on the diagnosis and management of paediatric patients with AA. A preliminary, evidence-based document issued by a group of paediatric haematologists was discussed, modified and approved during a series of {"}Consensus Conferences{"} according to procedures previously validated by the AIEOP Board. The guidelines highlight the importance of referring paediatric patients with AA to paediatric centres with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA; haematopoietic stem cell transplantation as first line therapy if an MSD is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CsA) if an MSD is not identified.",
keywords = "Acquired aplastic anemia, Childhood",
author = "Angelica Barone and Annunziata Lucarelli and Daniela Onofrillo and Federico Verzegnassi and Sonia Bonanomi and Daniela Longoni and Simone Cesaro and Chiara Cugno and Marco Zecca and Francesca Fioredda and Johanna Svahn and Carlo Dufour and Iori, {Anna Paola} and Saverio Ladogana and Matteo Maruzzi and Anna Locasciulli and Marina Lanciotti and Alessandra Macaluso and Rosalba Mandaglio and Nicoletta Marra and Giuseppe Menna and Baldo Martire and Notarangelo, {Lucia Dora} and Giovanni Palazzi and Marta Pillon and Ugo Ramenghi and Paola Saracco and Giovanna Russo and Fabio Timeus and Fabio Tucci and Piero Farruggia",
year = "2014",
language = "Italian",
volume = "33",
pages = "383--389",
journal = "Medico e Bambino",
issn = "1591-3090",
publisher = "Medico e Bambino",
number = "6",

}

TY - JOUR

T1 - Aplasie midollari acquisite in età pediatrica

T2 - Raccomandazioni diagnostico-terapeutiche

AU - Barone, Angelica

AU - Lucarelli, Annunziata

AU - Onofrillo, Daniela

AU - Verzegnassi, Federico

AU - Bonanomi, Sonia

AU - Longoni, Daniela

AU - Cesaro, Simone

AU - Cugno, Chiara

AU - Zecca, Marco

AU - Fioredda, Francesca

AU - Svahn, Johanna

AU - Dufour, Carlo

AU - Iori, Anna Paola

AU - Ladogana, Saverio

AU - Maruzzi, Matteo

AU - Locasciulli, Anna

AU - Lanciotti, Marina

AU - Macaluso, Alessandra

AU - Mandaglio, Rosalba

AU - Marra, Nicoletta

AU - Menna, Giuseppe

AU - Martire, Baldo

AU - Notarangelo, Lucia Dora

AU - Palazzi, Giovanni

AU - Pillon, Marta

AU - Ramenghi, Ugo

AU - Saracco, Paola

AU - Russo, Giovanna

AU - Timeus, Fabio

AU - Tucci, Fabio

AU - Farruggia, Piero

PY - 2014

Y1 - 2014

N2 - Acquired Aplastic Anaemia (AA) is a rare heterogeneous disease characterized by pancytopoenia and hypoplastic bone marrow. The incidence is 2-3 millions per year (all age groups) in Europe, but is higher in East Asia. The pathogenesis of AA is complex and involves haematopoietic stem cell/progenitor cell deficiencies and autoimmune mechanism. Survival in severe aplastic anaemia (SAA) has markedly improved in the past 2 decades because of advances in haematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA haematopoietic stem cell transplant (HSCT) from a matched sibling donor (MSD) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. The objective of this guideline is to provide healthcare professionals with clear guidance on the diagnosis and management of paediatric patients with AA. A preliminary, evidence-based document issued by a group of paediatric haematologists was discussed, modified and approved during a series of "Consensus Conferences" according to procedures previously validated by the AIEOP Board. The guidelines highlight the importance of referring paediatric patients with AA to paediatric centres with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA; haematopoietic stem cell transplantation as first line therapy if an MSD is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CsA) if an MSD is not identified.

AB - Acquired Aplastic Anaemia (AA) is a rare heterogeneous disease characterized by pancytopoenia and hypoplastic bone marrow. The incidence is 2-3 millions per year (all age groups) in Europe, but is higher in East Asia. The pathogenesis of AA is complex and involves haematopoietic stem cell/progenitor cell deficiencies and autoimmune mechanism. Survival in severe aplastic anaemia (SAA) has markedly improved in the past 2 decades because of advances in haematopoietic stem cell transplantation, immunosuppressive and biologic drugs, and supportive care. In SAA haematopoietic stem cell transplant (HSCT) from a matched sibling donor (MSD) is the treatment of choice. If a MSD is not available, the options include immunosuppressive therapy (IST) or unrelated donor HSCT. The objective of this guideline is to provide healthcare professionals with clear guidance on the diagnosis and management of paediatric patients with AA. A preliminary, evidence-based document issued by a group of paediatric haematologists was discussed, modified and approved during a series of "Consensus Conferences" according to procedures previously validated by the AIEOP Board. The guidelines highlight the importance of referring paediatric patients with AA to paediatric centres with long experience in diagnosis, differential diagnosis, management, supportive care and follow-up of AA; haematopoietic stem cell transplantation as first line therapy if an MSD is identified; the use of first-line IST containing horse anti-thymocyte globulin and cyclosporine A (CsA) if an MSD is not identified.

KW - Acquired aplastic anemia

KW - Childhood

UR - http://www.scopus.com/inward/record.url?scp=84905500565&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84905500565&partnerID=8YFLogxK

M3 - Article

VL - 33

SP - 383

EP - 389

JO - Medico e Bambino

JF - Medico e Bambino

SN - 1591-3090

IS - 6

ER -