Literature DB >> 26052913

Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes.

R Peffault de Latour1, C Peters2, B Gibson3, B Strahm4, A Lankester5, C D de Heredia6, D Longoni7, F Fioredda8, F Locatelli9, I Yaniv10, J Wachowiak11, J Donadieu12, A Lawitschka2, M Bierings13, M Wlodarski14, S Corbacioglu15, S Bonanomi16, S Samarasinghe17, T Leblanc18, C Dufour19, J-H Dalle18,20.   

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential to cure patients with an inherited bone marrow failure syndrome (IBMFS). However, the procedure involves the risk of treatment-related mortality and may be associated with significant early and late morbidity. For these reasons, the benefits should be carefully weighed against the risks. IBMFS are rare, whereas case reports and small series in the literature illustrate highly heterogeneous practices in terms of indications for HSCT, timing, stem cell source and conditioning regimens. A consensus meeting was therefore held in Vienna in September 2012 on behalf of the European Group for Blood and Marrow Transplantation to discuss HSCT in the setting of IBMFS. This report summarizes the recommendations from this expert panel, including indications for HSCT, timing, stem cell source and conditioning regimen.

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Year:  2015        PMID: 26052913     DOI: 10.1038/bmt.2015.117

Source DB:  PubMed          Journal:  Bone Marrow Transplant        ISSN: 0268-3369            Impact factor:   5.483


  39 in total

1.  Bone marrow versus peripheral blood as the stem cell source for sibling transplants in acquired aplastic anemia: survival advantage for bone marrow in all age groups.

Authors:  Andrea Bacigalupo; Gérard Socié; Hubert Schrezenmeier; Andre Tichelli; Anna Locasciulli; Monika Fuehrer; Antonio M Risitano; Carlo Dufour; Jakob R Passweg; Rosi Oneto; Mahmoud Aljurf; Catherine Flynn; Valerie Mialou; Rose Marie Hamladji; Judith C W Marsh
Journal:  Haematologica       Date:  2012-02-07       Impact factor: 9.941

2.  Haematopoietic cell transplantation for acute leukaemia and advanced myelodysplastic syndrome in Fanconi anaemia.

Authors:  Richard Mitchell; John E Wagner; Betsy Hirsch; Todd E DeFor; Heather Zierhut; Margaret L MacMillan
Journal:  Br J Haematol       Date:  2013-10-30       Impact factor: 6.998

3.  Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia.

Authors:  Mary Eapen; Jennifer Le Rademacher; Joseph H Antin; Richard E Champlin; Jeanette Carreras; Joseph Fay; Jakob R Passweg; Jakub Tolar; Mary M Horowitz; Judith C W Marsh; H Joachim Deeg
Journal:  Blood       Date:  2011-06-15       Impact factor: 22.113

Review 4.  How I treat Diamond-Blackfan anemia.

Authors:  Adrianna Vlachos; Ellen Muir
Journal:  Blood       Date:  2010-07-22       Impact factor: 22.113

5.  Cyclosporin A and short-term methotrexate versus cyclosporin A as graft versus host disease prophylaxis in patients with severe aplastic anemia given allogeneic bone marrow transplantation from an HLA-identical sibling: results of a GITMO/EBMT randomized trial.

Authors:  F Locatelli; B Bruno; M Zecca; M T Van-Lint; S McCann; W Arcese; S Dallorso; P Di Bartolomeo; F Fagioli; A Locasciulli; M Lawler; A Bacigalupo
Journal:  Blood       Date:  2000-09-01       Impact factor: 22.113

6.  HLA-matched related-donor HSCT in Fanconi anemia patients conditioned with cyclophosphamide and fludarabine.

Authors:  Lina Benajiba; Clementine Salvado; Jean-Hugues Dalle; Charlotte Jubert; Claire Galambrun; Jean Soulier; Gerard Socié; Regis Peffault de Latour
Journal:  Blood       Date:  2015-01-08       Impact factor: 22.113

7.  Analysis of risk factors for myelodysplasias, leukemias and death from infection among patients with congenital neutropenia. Experience of the French Severe Chronic Neutropenia Study Group.

Authors:  Jean Donadieu; Thierry Leblanc; Brigitte Bader Meunier; Mohamed Barkaoui; Odile Fenneteau; Yves Bertrand; Micheline Maier-Redelsperger; Marguerite Micheau; Jean Louis Stephan; Noel Phillipe; Pierre Bordigoni; Annie Babin-Boilletot; Philippe Bensaid; Anne Marie Manel; Etienne Vilmer; Isabelle Thuret; Stephane Blanche; Eliane Gluckman; Alain Fischer; Françoise Mechinaud; Bertrand Joly; Thierry Lamy; Olivier Hermine; Bruno Cassinat; Christine Bellanné-Chantelot; Christine Chomienne
Journal:  Haematologica       Date:  2005-01       Impact factor: 9.941

8.  Hematopoietic stem cell transplantation in severe congenital neutropenia: experience of the French SCN register.

Authors:  C Ferry; M Ouachée; T Leblanc; G Michel; A Notz-Carrére; R Tabrizi; T Flood; P Lutz; A Fischer; E Gluckman; J Donadieu
Journal:  Bone Marrow Transplant       Date:  2005-01       Impact factor: 5.483

9.  Unusual complications after bone marrow transplantation for dyskeratosis congenita.

Authors:  V Rocha; A Devergie; G Socié; P Ribaud; H Espérou; N Parquet; E Gluckman
Journal:  Br J Haematol       Date:  1998-10       Impact factor: 6.998

10.  Hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: experience of the French neutropenia registry.

Authors:  J Donadieu; G Michel; E Merlin; P Bordigoni; B Monteux; B Beaupain; G Leverger; J P Laporte; O Hermine; A Buzyn; Y Bertrand; J L Casanova; T Leblanc; E Gluckman; A Fischer; J L Stephan
Journal:  Bone Marrow Transplant       Date:  2005-11       Impact factor: 5.483

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  19 in total

1.  Old and new faces of neutropenia in children.

Authors:  Carlo Dufour; Maurizio Miano; Francesca Fioredda
Journal:  Haematologica       Date:  2016-07       Impact factor: 9.941

2.  Secreted protein Del-1 regulates myelopoiesis in the hematopoietic stem cell niche.

Authors:  Ioannis Mitroulis; Lan-Sun Chen; Rashim Pal Singh; Ioannis Kourtzelis; Matina Economopoulou; Tetsuhiro Kajikawa; Maria Troullinaki; Athanasios Ziogas; Klara Ruppova; Kavita Hosur; Tomoki Maekawa; Baomei Wang; Pallavi Subramanian; Torsten Tonn; Panayotis Verginis; Malte von Bonin; Manja Wobus; Martin Bornhäuser; Tatyana Grinenko; Marianna Di Scala; Andres Hidalgo; Ben Wielockx; George Hajishengallis; Triantafyllos Chavakis
Journal:  J Clin Invest       Date:  2017-08-28       Impact factor: 14.808

Review 3.  Inherited bone marrow failure syndromes: considerations pre- and posttransplant.

Authors:  Blanche P Alter
Journal:  Blood       Date:  2017-11-23       Impact factor: 22.113

4.  Extended clinical and genetic spectrum associated with biallelic RTEL1 mutations.

Authors:  Fabien Touzot; Laetitia Kermasson; Laurent Jullien; Despina Moshous; Christelle Ménard; Aydan Ikincioğullari; Figen Doğu; Sinan Sari; Vannina Giacobbi-Milet; Amos Etzioni; Jean Soulier; Arturo Londono-Vallejo; Alain Fischer; Isabelle Callebaut; Jean-Pierre de Villartay; Thierry Leblanc; Caroline Kannengiesser; Patrick Revy
Journal:  Blood Adv       Date:  2016-11-22

5.  Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia.

Authors:  Brigitte Strahm; Felicia Loewecke; Charlotte M Niemeyer; Michael Albert; Marc Ansari; Peter Bader; Yves Bertrand; Birgit Burkhardt; Lydie M Da Costa; Alina Ferster; Alexandra Fischer; Tayfun Güngör; Bernd Gruhn; Ina Hainmann; Friedrich Kapp; Peter Lang; Ingo Müller; Ansgar Schulz; Amina Szvetnik; Marcin Wlodarski; Peter Noellke; Thierry Leblanc; Jean-Hugues Dalle
Journal:  Blood Adv       Date:  2020-04-28

Review 6.  Old and new tools in the clinical diagnosis of inherited bone marrow failure syndromes.

Authors:  Allison H West; Jane E Churpek
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2017-12-08

Review 7.  Inherited bone marrow failure syndromes: considerations pre- and posttransplant.

Authors:  Blanche P Alter
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2017-12-08

Review 8.  Transplantation for bone marrow failure: current issues.

Authors:  Régis Peffault de Latour
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2016-12-02

Review 9.  Discussing and managing hematologic germ line variants.

Authors:  Wendy Kohlmann; Joshua D Schiffman
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2016-12-02

10.  Improvement of overall survival after allogeneic hematopoietic stem cell transplantation for children and adolescents: a three-decade experience of a single institution.

Authors:  E Brissot; F Rialland; X Cahu; M Strullu; N Corradini; C Thomas; N Blin; X Rialland; E Thebaud; P Chevallier; P Moreau; N Milpied; J L Harousseau; F Mechinaud; M Mohty
Journal:  Bone Marrow Transplant       Date:  2015-12-07       Impact factor: 5.483

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