Skip to main content
Key Specialties
Cardiology Diabetology Endocrinology Gastroenterology Geriatrics and Gerontology Gynecology and Obstetrics Hematology Infectious Disease Internal Medicine Nephrology Neurology Oncology Pediatrics Respiratory Medicine Rheumatology Surgery
Congress Coverage
2024 ACC Congress 2023 ESMO Congress 2023 EASD Congress 2023 ERS Congress 2023 ESC Congress 2023 EHA Congress 2023 ASCO Annual Meeting
Clinical Collections
Advances in Alzheimer’s

Keynote webinar | Spotlight on sleep in brain health

LIVE: Thursday 2nd May 2024, 18:00-19:30 (CEST)

Quality sleep is essential for health. But what happens to our brains when sleep patterns are disturbed? Join our experts to explore the interplay between sleep disruption and neurological diseases, and the questions that you need to be asking your patients to help you prevent the harmful effects of sleep deprivation.
ADVANCED SEARCH
Log in
Top
International Journal of Hematology
Published in: International Journal of Hematology 4/2016

01-04-2016 | Progress in Hematology

Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes

Authors: Jean-Hugues Dalle, Régis Peffault de Latour

Published in: International Journal of Hematology | Issue 4/2016

Login to get access

Abstract

Inherited bone marrow failure (IBMF) syndromes are a heterogeneous group of rare hematological disorders characterized by the impairment of hematopoiesis, which harbor specific clinical presentations and pathogenic mechanisms. Some of these syndromes may progress through clonal evolution, myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML). Most prominent are failures of DNA repair such as Fanconi Anemia and much rarer failure of ribosomal apparatus, e.g., Diamond Blackfan Anemia or of telomere elongation such as dyskeratosis congenita. In these congenital disorders, hematopoietic stem cell transplantation (HSCT) is often a consideration. However, HSCT will not correct the underlying disease and possible co-existing extra-medullary (multi)-organ defects, but will improve BMF. Indications as well as transplantation characteristics are most of the time controversial in this setting because of the rarity of reported cases. The present paper proposes a short overview of current practices.
Literature
1.
Ghemlas I, Li H, Zlateska B, Klaassen R, Fernandez CV, Yanofsky RA, Wu J, Pastore Y, Silva M, Lipton JH, et al. Improving diagnostic precision, care and syndrome definitions using comprehensive next-generation sequencing for the inherited bone marrow failure syndromes. J Med Genet. 2015;52:575–84.CrossRefPubMed
2.
Zhang MY, Keel SB, Walsh T, Lee MK, Gulsuner S, Watts AC, Pritchard CC, Salipante SJ, Jeng MR, Hofmann I, et al. Genomic analysis of bone marrow failure and myelodysplastic syndromes reveals phenotypic and diagnostic complexity. Haematologica. 2015;100:42–8.CrossRefPubMedPubMedCentral
3.
4.
Ameziane N, Sie D, Dentro S, Ariyurek Y, Kerkhoven L, Joenje H, Dorsman JC, Ylstra B, Gille JJ, Sistermans EA, de Winter JP. Diagnosis of Fanconi anemia: mutation analysis by next-generation sequencing. Anemia. 2012;2012:132856.CrossRefPubMedPubMedCentral
5.
Bogliolo M, Surralles J. Fanconi anemia: a model disease for studies on human genetics and advanced therapeutics. Curr Opin Genet Dev. 2015;33:32–40.CrossRefPubMed
6.
Gluckman E, Auerbach AD, Horowitz MM, Sobocinski KA, Ash RC, Bortin MM, Butturini A, Camitta BM, Champlin RE, Friedrich W, et al. Bone marrow transplantation for Fanconi anemia. Blood. 1995;86:2856–62.PubMed
7.
Guardiola P, Pasquini R, Dokal I, Ortega JJ, van Weel-Sipman M, Marsh JC, Ball SE, Locatelli F, Vermylen C, Skinner R, et al. Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood. 2000;95:422–9.PubMed
8.
Locatelli F, Zecca M, Pession A, Morreale G, Longoni D, Di Bartolomeo P, Porta F, Fagioli F, Nobili B, Bernardo ME, et al. The outcome of children with Fanconi anemia given hematopoietic stem cell transplantation and the influence of fludarabine in the conditioning regimen: a report from the Italian pediatric group. Haematologica. 2007;92:1381–8.CrossRefPubMed
9.
Wagner JE, Eapen M, MacMillan ML, Harris RE, Pasquini R, Boulad F, Zhang MJ, Auerbach AD. Unrelated donor bone marrow transplantation for the treatment of Fanconi anemia. Blood. 2007;109:2256–62.CrossRefPubMedPubMedCentral
10.
Deeg HJ, Socie G, Schoch G, Henry-Amar M, Witherspoon RP, Devergie A, Sullivan KM, Gluckman E, Storb R. Malignancies after marrow transplantation for aplastic anemia and Fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood. 1996;87:386–92.PubMed
11.
Malric A, Defachelles AS, Leblanc T, Lescoeur B, Lacour B, Peuchmaur M, Maurage CA, Pierron G, Guillemot D, d’Enghien CD, et al. Fanconi anemia and solid malignancies in childhood: a national retrospective study. Pediatr Blood Cancer. 2015;62:463–70.CrossRefPubMed
12.
Masserot C, Peffault de Latour R, Rocha V, Leblanc T, Rigolet A, Pascal F, Janin A, Soulier J, Gluckman E, Socie G. Head and neck squamous cell carcinoma in 13 patients with Fanconi anemia after hematopoietic stem cell transplantation. Cancer. 2008;113:3315–22.CrossRefPubMed
13.
Gluckman E, Devergie A, Dutreix J. Radiosensitivity in Fanconi anaemia: application to the conditioning regimen for bone marrow transplantation. Br J Haematol. 1983;54:431–40.CrossRefPubMed
14.
Medeiros C, Zanis-Neto J, Pasquini R. Bone marrow transplantation for patients with Fanconi anemia: reduced doses of cyclophosphamide without irradiation as conditioning. Bone Marrow Transplant. 1999;24:849–52.CrossRefPubMed
15.
de la Fuente J, Reiss S, McCloy M, Vulliamy T, Roberts IA, Rahemtulla A, Dokal I. Non-TBI stem cell transplantation protocol for Fanconi anaemia using HLA-compatible sibling and unrelated donors. Bone Marrow Transplant. 2003;32:653–6.CrossRefPubMed
16.
Guardiola P, Socie G, Li X, Ribaud P, Devergie A, Esperou H, Richard P, Traineau R, Janin A, Gluckman E. Acute graft-versus-host disease in patients with Fanconi anemia or acquired aplastic anemia undergoing bone marrow transplantation from HLA-identical sibling donors: risk factors and influence on outcome. Blood. 2004;103:73–7.CrossRefPubMed
17.
MacMillan ML, DeFor TE, Young JA, Dusenbery KE, Blazar BR, Slungaard A, Zierhut H, Weisdorf DJ, Wagner JE. Alternative donor hematopoietic cell transplantation for Fanconi anemia. Blood. 2015;125:3798–804.CrossRefPubMed
18.
Shimada A, Takahashi Y, Muramatsu H, Hama A, Ismael O, Narita A, Sakaguchi H, Doisaki S, Nishio N, Tanaka M, et al. Excellent outcome of allogeneic bone marrow transplantation for Fanconi anemia using fludarabine-based reduced-intensity conditioning regimen. Int J Hematol. 2012;95:675–9.CrossRefPubMed
19.
Chaudhury S, Auerbach AD, Kernan NA, Small TN, Prockop SE, Scaradavou A, Heller G, Wolden S, O’Reilly RJ, Boulad F. Fludarabine-based cytoreductive regimen and T-cell-depleted grafts from alternative donors for the treatment of high-risk patients with Fanconi anaemia. Br J Haematol. 2008;140:644–55.CrossRefPubMed
20.
de Peffault Latour R, Porcher R, Dalle JH, Aljurf M, Korthof ET, Svahn J, Willemze R, Barrenetxea C, Mialou V, Soulier J, et al. Allogeneic hematopoietic stem cell transplantation in Fanconi anemia: the European Group for Blood and Marrow Transplantation experience. Blood. 2013;122:4279–86.CrossRef
21.
Stepensky P, Shapira MY, Balashov D, Trakhtman P, Skorobogatova E, Rheingold L, Brooks R, Revel-Vilk S, Weintraub M, Stein J, et al. Bone marrow transplantation for Fanconi anemia using fludarabine-based conditioning. Biol Blood Marrow Transplant. 2011;17:1282–8.CrossRefPubMed
22.
Yabe H, Inoue H, Matsumoto M, Hamanoue S, Koike T, Ishiguro H, Koike H, Suzuki K, Kato S, Kojima S, et al. Allogeneic haematopoietic cell transplantation from alternative donors with a conditioning regimen of low-dose irradiation, fludarabine and cyclophosphamide in Fanconi anaemia. Br J Haematol. 2006;134:208–12.CrossRefPubMed
23.
MacMillan ML, Wagner JE. Haematopoeitic cell transplantation for Fanconi anaemia—when and how? Br J Haematol. 2010;149:14–21.CrossRefPubMed
24.
Gluckman E, Ruggeri A, Volt F, Cunha R, Boudjedir K, Rocha V. Milestones in umbilical cord blood transplantation. Br J Haematol. 2011;154:441–7.CrossRefPubMed
25.
Bacigalupo A, Socie G, Schrezenmeier H, Tichelli A, Locasciulli A, Fuehrer M, Risitano AM, Dufour C, Passweg JR, Oneto R, et al. 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. Haematologica. 2012;97:1142–8.CrossRefPubMedPubMedCentral
26.
Eapen M, Le Rademacher J, Antin JH, Champlin RE, Carreras J, Fay J, Passweg JR, Tolar J, Horowitz MM, Marsh JC, Deeg HJ. Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia. Blood. 2011;118:2618–21.CrossRefPubMedPubMedCentral
27.
Schrezenmeier H, Passweg JR, Marsh JC, Bacigalupo A, Bredeson CN, Bullorsky E, Camitta BM, Champlin RE, Gale RP, Fuhrer M, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA-matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood. 2007;110:1397–400.CrossRefPubMedPubMedCentral
28.
29.
Talbot A, Peffault de Latour R, Raffoux E, Buchbinder N, Vigouroux S, Milpied N, Leblanc T, Soulier J, Michallet M, Socie G. Sequential treatment for allogeneic hematopoietic stem cell transplantation in Fanconi anemia with acute myeloid leukemia. Haematologica. 2014;99:e199–200.CrossRefPubMedPubMedCentral
30.
Kohli-Kumar M, Morris C, DeLaat C, Sambrano J, Masterson M, Mueller R, Shahidi NT, Yanik G, Desantes K, Friedman DJ, et al. Bone marrow transplantation in Fanconi anemia using matched sibling donors. Blood. 1994;84:2050–4.PubMed
31.
Kostmann R. Infantile genetic agranulocytosis; agranulocytosis infantilis hereditaria. Acta Paediatr Suppl. 1956;45:1–78.CrossRefPubMed
32.
Bellanne-Chantelot C, Clauin S, Leblanc T, Cassinat B, Rodrigues-Lima F, Beaufils S, Vaury C, Barkaoui M, Fenneteau O, Maier-Redelsperger M, et al. Mutations in the ELA2 gene correlate with more severe expression of neutropenia: a study of 81 patients from the French Neutropenia Register. Blood. 2004;103:4119–25.CrossRefPubMed
33.
Germeshausen M, Zeidler C, Stuhrmann M, Lanciotti M, Ballmaier M, Welte K. Digenic mutations in severe congenital neutropenia. Haematologica. 2010;95:1207–10.CrossRefPubMedPubMedCentral
34.
Person RE, Li FQ, Duan Z, Benson KF, Wechsler J, Papadaki HA, Eliopoulos G, Kaufman C, Bertolone SJ, Nakamoto B, et al. Mutations in proto-oncogene GFI1 cause human neutropenia and target ELA2. Nat Genet. 2003;34:308–12.CrossRefPubMedPubMedCentral
35.
Sayour EJ, Carter C, Eldjerou L. A novel ELANE gene mutation in a patient with severe congenital neutropenia and intermittent thrombocytopenia. Pediatr Blood Cancer. 2015;62:1870–1.CrossRefPubMed
36.
Touw IP. Game of clones: the genomic evolution of severe congenital neutropenia. Hematology Am Soc Hematol Educ Program. 2015;2015:1–7.PubMed
37.
Dale DC, Cottle TE, Fier CJ, Bolyard AA, Bonilla MA, Boxer LA, Cham B, Freedman MH, Kannourakis G, Kinsey SE, et al. Severe chronic neutropenia: treatment and follow-up of patients in the Severe Chronic Neutropenia International Registry. Am J Hematol. 2003;72:82–93.CrossRefPubMed
38.
Bonilla MA, Gillio AP, Ruggeiro M, Kernan NA, Brochstein JA, Abboud M, Fumagalli L, Vincent M, Gabrilove JL, Welte K, et al. Effects of recombinant human granulocyte colony-stimulating factor on neutropenia in patients with congenital agranulocytosis. N Engl J Med. 1989;320:1574–80.CrossRefPubMed
39.
Dalle JH, Donadieu J, Paillard C, Rialland F, Schneider P, Sicre De Fontbrune F, Hichri Y, Neven B, Yakoub-Agha I, Sfgm TC. [SFGM-TC recommendation on indications for allogeneic stem cell transplantation in children with congenital neutropenia]. Pathol Biol (Paris). 2014;62:209–11.CrossRef
40.
Ferry C, Ouachee M, Leblanc T, Michel G, Notz-Carrere A, Tabrizi R, Flood T, Lutz P, Fischer A, Gluckman E, Donadieu J. Hematopoietic stem cell transplantation in severe congenital neutropenia: experience of the French SCN register. Bone Marrow Transplant. 2005;35:45–50.CrossRefPubMed
41.
Fioredda F, Iacobelli S, van Biezen A, Gaspar B, Ancliff P, Donadieu J, Aljurf M, Peters C, Calvillo M, Matthes-Martin S, et al. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation. Blood. 2015;126:1885–92.CrossRefPubMed
42.
Oshima K, Hanada R, Kobayashi R, Kato K, Nagatoshi Y, Tabuchi K, Kato S. Hematopoietic Stem Cell Transplantation Committee of the Japanese Society of Pediatric H: hematopoietic stem cell transplantation in patients with severe congenital neutropenia: an analysis of 18 Japanese cases. Pediatr Transplant. 2010;14:657–63.CrossRefPubMed
43.
Donadieu J, Leblanc T, Bader Meunier B, Barkaoui M, Fenneteau O, Bertrand Y, Maier-Redelsperger M, Micheau M, Stephan JL, Phillipe N, et al. 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. Haematologica. 2005;90:45–53.PubMed
44.
Osone S, Imamura T, Fukushima-Nakase Y, Kitamura-Masaki A, Kanai S, Imai T, Imashuku S, Kuroda H. Case reports of severe congenital neutropenia treated with unrelated cord blood transplantation with reduced-intensity conditioning. J Pediatr Hematol Oncol. 2016;38:49–52.CrossRefPubMed
45.
Anderson JE, Gooley TA, Schoch G, Anasetti C, Bensinger WI, Clift RA, Hansen JA, Sanders JE, Storb R, Appelbaum FR. Stem cell transplantation for secondary acute myeloid leukemia: evaluation of transplantation as initial therapy or following induction chemotherapy. Blood. 1997;89:2578–85.PubMed
46.
Cassinat B, Bellanne-Chantelot C, Notz-Carrere A, Menot ML, Vaury C, Micheau M, Bader-Meunier B, Perel Y, Leblanc T, Donadieu J, Chomienne C. Screening for G-CSF receptor mutations in patients with secondary myeloid or lymphoid transformation of severe congenital neutropenia. A report from the French neutropenia register. Leukemia. 2004;18:1553–5.CrossRefPubMed
47.
Germeshausen M, Ballmaier M, Schulze H, Welte K, Flohr T, Beiske K, Storm-Mathisen I, Abrahamsen TG. Granulocyte colony-stimulating factor receptor mutations in a patient with acute lymphoblastic leukemia secondary to severe congenital neutropenia. Blood. 2001;97:829–30.CrossRefPubMed
48.
Ebihara Y, Ishikawa K, Mochizuki S, Tanaka R, Manabe A, Iseki T, Maekawa T, Tsuji K. Allogeneic stem cell transplantation for patients with acute myeloid leukaemia developing from severe congenital neutropenia. Br J Haematol. 2014;164:459–61.CrossRefPubMed
49.
50.
Boocock GR, Morrison JA, Popovic M, Richards N, Ellis L, Durie PR, Rommens JM. Mutations in SBDS are associated with Shwachman-Diamond syndrome. Nat Genet. 2003;33:97–101.CrossRefPubMed
51.
Donadieu J, Fenneteau O, Beaupain B, Beaufils S, Bellanger F, Mahlaoui N, Lambilliotte A, Aladjidi N, Bertrand Y, Mialou V, et al. Classification of and risk factors for hematologic complications in a French national cohort of 102 patients with Shwachman-Diamond syndrome. Haematologica. 2012;97:1312–9.CrossRefPubMedPubMedCentral
52.
Dror Y, Donadieu J, Koglmeier J, Dodge J, Toiviainen-Salo S, Makitie O, Kerr E, Zeidler C, Shimamura A, Shah N, et al. Draft consensus guidelines for diagnosis and treatment of Shwachman-Diamond syndrome. Ann NY Acad Sci. 2011;1242:40–55.CrossRefPubMed
53.
Sauer M, Zeidler C, Meissner B, Rehe K, Hanke A, Welte K, Lohse P, Sykora KW. Substitution of cyclophosphamide and busulfan by fludarabine, treosulfan and melphalan in a preparative regimen for children and adolescents with Shwachman-Diamond syndrome. Bone Marrow Transplant. 2007;39:143–7.CrossRefPubMed
54.
Fagioli F, Quarello P, Zecca M, Lanino E, Corti P. avre F, Ripaldi M, Ramenghi U, Locatelli F, Prete A: haematopoietic stem cell transplantation for Diamond Blackfan anemia: a report from the Italian association of paediatric haematology and oncology registry. Br J Haematol. 2014;165:673–81.CrossRefPubMed
55.
Vlachos A, Muir E. How I treat Diamond-Blackfan anemia. Blood. 2010;116:3715–23.PubMed
56.
Ballmaier M, Germeshausen M. Congenital amegakaryocytic thrombocytopenia: clinical presentation, diagnosis, and treatment. Semin Thromb Hemost. 2011;37:673–81.CrossRefPubMed
57.
Dalle JH, Fahd M. Allogeneic stem cell transplantation in amegacaryocytosis: results of a retrospective study in EBMT centers. Biol Blood Marrow Transplant. 2014;20:S81–2.CrossRef
58.
Mahadeo KM, Tewari P, Parikh SH, Driscoll TA, Page K, Martin PL, Kurtzberg J, Prasad VK. Durable engraftment and correction of hematological abnormalities in children with congenital amegakaryocytic thrombocytopenia following myeloablative umbilical cord blood transplantation. Pediatr Transplant. 2015;19:753–7.CrossRefPubMed
59.
de la Fuente J, Dokal I. Dyskeratosis congenita: advances in the understanding of the telomerase defect and the role of stem cell transplantation. Pediatr Transplant. 2007;11:584–94.CrossRefPubMed
60.
Dror Y, Freedman MH, Leaker M, Verbeek J, Armstrong CA, Saunders FE, Doyle JJ. Low-intensity hematopoietic stem-cell transplantation across human leucocyte antigen barriers in dyskeratosis congenita. Bone Marrow Transplant. 2003;31:847–50.CrossRefPubMed
61.
Rocha V, Devergie A, Socie G, Ribaud P, Esperou H, Parquet N, Gluckman E. Unusual complications after bone marrow transplantation for dyskeratosis congenita. Br J Haematol. 1998;103:243–8.CrossRefPubMed
62.
Gadalla SM, Sales-Bonfim C, Carreras J, Alter BP, Antin JH, Ayas M, Bodhi P, Davis J, Davies SM, Deconinck E, et al. Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenita. Biol Blood Marrow Transplant. 2013;19:1238–43.CrossRefPubMedPubMedCentral
63.
Bizzetto R, Bonfim C, Rocha V, Socie G, Locatelli F, Chan K, Ramirez O, Stein J, Nabhan S, Miranda E, et al. Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia. Haematologica. 2011;96:134–41.CrossRefPubMedPubMedCentral
64.
Dietz AC, Orchard PJ, Baker KS, Giller RH, Savage SA, Alter BP, Tolar J. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita. Bone Marrow Transplant. 2011;46:98–104.CrossRefPubMed
65.
Nishio N, Takahashi Y, Ohashi H, Doisaki S, Muramatsu H, Hama A, Shimada A, Yagasaki H, Kojima S. Reduced-intensity conditioning for alternative donor hematopoietic stem cell transplantation in patients with dyskeratosis congenita. Pediatr Transplant. 2011;15:161–6.PubMed
66.
Vuong LG, Hemmati PG, Neuburger S, Terwey TH, Vulliamy T, Dokal I, le Coutre P, Dorken B, Arnold R. Reduced-intensity conditioning using fludarabine and antithymocyte globulin alone allows stable engraftment in a patient with dyskeratosis congenita. Acta Haematol. 2010;124:200–3.CrossRefPubMed
67.
68.
Amarasinghe K, Dalley C, Dokal I, Laurie A, Gupta V, Marsh J. Late death after unrelated-BMT for dyskeratosis congenita following conditioning with alemtuzumab, fludarabine and melphalan. Bone Marrow Transplant. 2007;40:913–4.CrossRefPubMed
69.
Brazzola P, Duval M, Fournet JC, Gauvin F, Dalle JH, Champagne J, Champagne MA. Fatal diffuse capillaritis after hematopoietic stem-cell transplantation for dyskeratosis congenita despite low-intensity conditioning regimen. Bone Marrow Transplant. 2005;36:1103–5 (author reply 1105).CrossRefPubMed
70.
Ayas M, Nassar A, Hamidieh AA, Kharfan-Dabaja M, Othman TB, Elhaddad A, Seraihy A, Hussain F, Alimoghaddam K, Ladeb S, et al. Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure. Bone Marrow Transplant. 2013;48:1168–72.CrossRefPubMed
Metadata
Title
Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes
Authors
Jean-Hugues Dalle
Régis Peffault de Latour
Publication date
01-04-2016
Publisher
Springer Japan
Published in
International Journal of Hematology / Issue 4/2016
Print ISSN: 0925-5710
Electronic ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-016-1951-0

Other articles of this Issue 4/2016

International Journal of Hematology 4/2016 Go to the issue

Images in Hematology

Leukemic mastopathy

Original Article

Prior hypomethylating agent use lacks impact on clinical outcome in patients with secondary acute myeloid leukemia arising from myelodysplastic syndromes treated with standard induction chemotherapy

Special Note

Commentary: How do haematopoietic cell transplants cure leukaemia?

Original Article

Soluble interleukin-2 receptor index predicts the development of acute graft-versus-host disease after allogeneic hematopoietic stem cell transplantation from unrelated donors

Original Article

Residual disease detected by multidimensional flow cytometry shows prognostic significance in childhood acute myeloid leukemia with intermediate cytogenetics and negative FLT3-ITD: a report from the Tokyo Children’s Cancer Study Group

Progress in Hematology

Evolution of myeloid leukemia in children with Down syndrome
Webinar | 19-02-2024 | 17:30 (CET)

Keynote webinar | Spotlight on antibody–drug conjugates in cancer

Watch now

Antibody–drug conjugates (ADCs) are novel agents that have shown promise across multiple tumor types. Explore the current landscape of ADCs in breast and lung cancer with our experts, and gain insights into the mechanism of action, key clinical trials data, existing challenges, and future directions.

Dr. Véronique Diéras
Prof. Fabrice Barlesi
Developed by: Springer Medicine
Image Credits