Biblio

Author Title [ Type(Asc)] Year
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Journal Article
Vallée TC, Glasmacher JS, Buchner H, Arkwright PD, Behrends U, Bondarenko A, Browning MJ, Buchbinder DK, Cattoni A, Chernyshova L, et al. Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity. Blood. 2024.
Lehmberg K, Albert MH, Beier R, Beutel K, Gruhn B, Kröger N, Meisel R, Schulz A, Stachel D, Woessmann W, et al. Treosulfan-based conditioning regimen for children and adolescents with hemophagocytic lymphohistiocytosis. Haematologica. 2013.
Hoeben BAW, Pazos M, Albert MH, Seravalli E, Bosman ME, Losert C, Boterberg T, Manapov F, Ospovat I, Milla SMico, et al. Towards homogenization of total body irradiation practices in pediatric patients across SIOPE affiliated centers a survey by the SIOPE radiation oncology working group. Radiother Oncol. 2020.
Egg D, Rump ICaroline, Mitsuiki N, Rojas-Restrepo J, Maccari M-E, Schwab C, Gabrysch A, Warnatz K, Goldacker S, Patiño V, et al. Therapeutic options for CTLA-4 Insufficiency. J Allergy Clin Immunol. 2021.
Felber M, Steward CG, Kentouche K, Fasth A, Wynn RF, Zeilhofer U, Haunerdinger V, Volkmer B, Prader S, Gruhn B, et al. Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis. Blood Adv. 2020;4(9):1998-2010.
Sahoo SS, Pastor VB, Goodings C, Voss RK, Kozyra EJ, Szvetnik A, Noellke P, Dworzak M, Stary J, Locatelli F, et al. Publisher Correction: Clinical evolution, genetic landscape and trajectories of clonal hematopoiesis in SAMD9/SAMD9L syndromes. Nat Med. 2021.
Wlodarski MW, Hirabayashi S, Pastor V, Stary J, Hasle H, Masetti R, Dworzak M, Schmugge M, van den Heuvel-Eibrink M, Ussowicz M, et al. Prevalence, clinical characteristics and prognosis of GATA2-related myelodysplastic syndromes (MDS) in children and adolescents. Blood. 2015.
von Asmuth EGj, Neven B, Albert MH, Mohseny AB, Schilham MW, Binder H, Putter H, Lankester AC. Predicting patient death after allogeneic SCT for inborn errors using machine learning (PREPAD): An EBMT IEWP study. Transplant Cell Ther. 2023.
Boehmer DFR, Koehler LM, Magg T, Metzger P, Rohlfs M, Ahlfeld J, Rack-Hoch A, Reiter K, Albert MH, Endres S, et al. A novel complete autosomal recessive STAT1 LOF variant causes immunodeficiency with hemophagocytic lymphohistiocytosis-like hyperinflammation. J Allergy Clin Immunol Pract. 2020.
Ghosh S, Albert MH, Hauck F, Hönig M, Schütz C, Schulz A, Speckmann C. [Newborn screening for severe combined immunodeficiencies (SCID) in Germany]. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2023.
Hoenig M, Roesler J, Seidel MG, Albert MH, Hauck F, Maecker-Kolhoff B, Eiz-Vesper B, Kleinschmidt K, Debatin K-M, Jacobsen E-M, et al. Matched Family Donor Lymphocyte Infusions as First Cellular Therapy for Patients with Severe Primary T-cell Deficiencies. Biol Blood Marrow Transplant. 2020.
Ferrua F, Cicalese MPia, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo MEster, Calbi V, Assanelli AAngelo, et al. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Lancet Haematol. 2019.
Fischer M, Olbrich P, Hadjadj J, Aumann V, Bakhtiar S, Barlogis V, von Bismarck P, Bloomfield MPhDMarké, Booth C, Buddingh EP, et al. JAK-inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID and EBMT IEWP retrospective study. J Allergy Clin Immunol. 2023.
Nishimura A, Uppuluri R, Raj R, Swaminathan VVellaicham, Cheng Y, Abu-Arja RF, Fu B, Laberko A, Albert MH, Hauck F, et al. An International Survey of Allogeneic Hematopoietic Cell Transplantation for X-Linked Agammaglobulinemia. J Clin Immunol. 2023.
Schuetz C, Gerke J, Ege MJohannes, Walter JEszter, Kusters M, Worth AJJ, Kanakry JA, Dimitrova D, Wolska-Kusnierz B, Chen K, et al. Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCT. Blood. 2022.
Albert MH, Slatter MA, Gennery AR, Güngör T, Bakunina K, Markovitch B, Hazelaar S, Sirait T, Courteille V, Aiuti A, et al. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis. Blood. 2022.
Ferrua F, Galimberti S, Courteille V, Slatter MAnne, Booth C, Moshous D, Neven B, Blanche S, Laberko A, Shcherbina A, et al. Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-SCETIDE-PIDTC Study. J Allergy Clin Immunol. 2019.
Albert MH, Sirait T, Eikema D-J, Bakunina K, Wehr C, Suarez F, Fox MLaura, Mahlaoui N, Gennery AR, Lankester AC, et al. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity, an EBMT IEWP study. Blood. 2022.
Vallée T, Schmid I, Gloning L, Bacova M, Ahrens J, Feuchtinger T, Klein C, Gaertner VD, Albert MH. Excellent outcome of stem cell transplantation for sickle cell disease. Ann Hematol. 2023.
Mekelenkamp H, de Vries M, Markovitch B, Sirait T, Pieterse AH, Bense J, Kleinschmidt K, Albert MH, Neven B, Corbacioglu S, et al. Decision making on HSCT in patients with hemoglobinopathies; an EBMT Pediatric Diseases Working Party and Inborn Errors Working Party scenario-based survey on physicians' perspectives. Bone Marrow Transplant. 2023.
Tsilifis C, Torppa T, Williams EJ, Albert MH, Hauck F, Soncini E, Kang E, Malech H, Schuetz C, von Bernuth H, et al. Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers. J Clin Immunol. 2023.
Pichler H, Lawitschka A, Glogova E, Willasch AM, von Luettichau I, Lehrnbecher T, Matthes-Martin S, Lang P, Bader P, Sykora KW, et al. Allogeneic hematopoietic stem cell transplantation from unrelated donors is associated with higher infection rates in children with acute lymphoblastic leukemia - A prospective international multicenter trial on behalf of the BFM-SG and the EBMT-PDWP. Am J Hematol. 2019.