Inborn Errors Working Party - EBMT Errors Working Party.pdf1 #EBMT20Virtual Haematopoietic Cell...
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www.ebmt.org #EBMT20Virtual 1 Haematopoietic Cell Transplantation in Chronic Granulomatous Disease: a Study on 712 Children and Adults Blood 2020 Chiesa R,. Wang J., Blok H.J., Hazelaar S., Neven B., Moshous D., Schulz A.S., Hoenig M., Hauck F., Al Seraihy A., Gozdzik J., Ljungman P.T., Lindemans C., Fernandes J., Kalwak K., Strahm B., Schanz U., Sedlacek P., Sykora K.W., Aksoylar S., Locatelli F., Stepensky P., Wynn R.F., Lum S.H., Zecca M., Porta F., Taskinen M.H., Gibson B.E., Matthes-Martin S., Karakukcu M., Hauri-Hohl M.M., Veys P., Gennery A.R., Lucchini G., Felber M., Albert M.H., Balashov D., Lankester A.C., Güngör T., Slatter M. Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP- SCETIDE-PIDTC Study J Allergy Clin Immunol 2019 Ferrua F., Galimberti S., Courteille V., Slatter M.A., Booth C., Moshous D., Neven B., Blanche S., Cavazzana M., Laberko A., Shcherbina A., Balashov D., Soncini E., Porta F., Al-Mousa H., Al-Saud B., Al-Dhekri H., Arnaout R., Formankova R., Bertrand Y., Lange A., Smart J., Wolska-Kusnierz B., Aquino V.M., Dvorak C.C., Fasth A., Fouyssac F., Heilmann C., Hoenig M., Schuetz C., Kelecic J., Bredius R.G.M., Lankester A.C., Lindemans C.A., Suarez F., Sullivan K.E., Albert M.H., Kalwak K., Barlogis V., Bhatia M., Bordon V., Czogala W., Alonso L., Dogu F., Gozdzik J., Ikinciogullari A., Krivan G., Ljungman P., Meyts I., Mustillo P., Smith A.R., Speckmann C., Sundin M., Keogh S.J., Shaw P.J., Boelens J.J., Schulz A.S., Sedlacek P., Veys P., Mahlaoui N., Janda A., Davies E.G., Fischer A., Cowan M.J., Gennery A.R. Hematopoietic Stem Cell Transplantation for Primary Immunodeficiencies Front Pediatr 2019 Gennery, A. R., Albert M. H., Slatter M. A., and Lankester A. Hematopoietic Cell Transplantation for MHC Class II Deficiency Front Pediatr 2019 Lum, S. H., Neven B., Slatter M. A., and Gennery, A. R. Conditioning perspectives for Primary Immunodeficiency Stem Cell Transplants Front Pediatr 2019 Shaw, P., Shizuru J., Hoenig M., and Veys P. Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism Front Pediatr 2019 Tan, E. Y., Boelens J. J., Jones S. A., and Wynn R. F. Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Immune Dysregulatory Disorders Front Pediatr 2019 Bakhtiar, S., Fekadu J., Seidel M. G., and Gambinieri E. Wiskott -Aldrich Syndrome (WAS) and Dedicator of Cytokinesis 8- (DOCK8) Deficiency Front Pediatr 2019 Albert, M. H., & Freeman A. F. The Evidence for Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Neutrophil Disorders: A Comprehensive Review by the Inborn Errors Working Party Group of the EBMT Front Pediatr 2019 Bakhtiar, S., Shadur, B., and Stepensky, P. Long Term Outcome and Immune Function After Hematopoietic Stem Cell Transplantation for Primary Immunodeficiency Front Pediatr 2019 Gennery, A. R., Lankester, A. Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and PDWP Working Parties Front Pediatr 2019 Greco R., Labopin M., Badoglio M., Veys P., Furtado Silva J.M., Abinun M., Gualandi F., Bornhauser M., Ciceri F., Saccardi R., Lankester A., Alexander T., Gennery A.R., Bader P., Farge D., Snowden J.A. Universal Newborn Screening for Severe Combined Immunodeficiency (SCID) Front Pediatr 2019 van der Burg M., Mahlaoui N., Gaspar H.B., Pai S-Y. Allogeneic HSCT in Adolescents and Young Adults With Primary Immunodeficiencies Front Pediatr 2019 Morris E.C., Albert M.H. Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis Front Pediatr 2019 Lehmberg K., Moshous D., Booth C. Arjan Lankester (chair) Leiden, the Netherlands Michael Albert (secretary/co-chair) Munich, Germany Inborn Errors Working Party Arjan Lankester Chair [email protected] Yvonne Wijkhuisen Secretary A. Lankester Y.W.Wijkhuisen[email protected] Michael Albert Secretary/Co-Chair [email protected] -muenchen.de Sheree Hazelaar EBMT Data manager [email protected] Recent Publications on behalf of IEWP Studies EBMT Registry Virtual IEWP events Future Events 2020 Contact IEWP Topic* Disease type Joint AYA PID PID Haplo in PID PID ICF Syndrome PID IFNg R1 R2 PID Allo in MLD PID Second Allo PID Mevalonate Kinase Deficiency PID CD27/CD70 deficiency PID IEWP – ESID * If you whish to participate in data collection for these studies, please contact: [email protected] Tuesday September 1 st IEWP session 11.00 – 12.00 Auditorium 4 Program IEWP session: ▪ 11.00 – 11.12 Landscape of GT and allogeneic Therapy in IEM: The next years - Robert Wynn ▪ 11.12 – 11.24 Therapeutic standards for patients with SCID- still a matter of discussion - Manfred Hönig ▪ 11.24 – 11.36 Induction therapy in Inborn Errors of Immunity before HSCT – Victoria Tesch ▪ 11.36 – 11.48 Inflammation and autoimmunity in PID: Does it matter for HSCT? - Bénédicte Neven ▪ 11.48 – 12.00 Discussion Significant Collaborations
Transcript of Inborn Errors Working Party - EBMT Errors Working Party.pdf1 #EBMT20Virtual Haematopoietic Cell...
www.ebmt.org #EBMT20Virtual1
Haematopoietic Cell Transplantation in Chronic Granulomatous Disease: a Study on 712 Children and
Adults Blood 2020
Chiesa R,. Wang J., Blok H.J., Hazelaar S., Neven B., Moshous D., Schulz A.S., Hoenig M., Hauck F., Al Seraihy
A., Gozdzik J., Ljungman P.T., Lindemans C., Fernandes J., Kalwak K., Strahm B., Schanz U., Sedlacek P., Sykora
K.W., Aksoylar S., Locatelli F., Stepensky P., Wynn R.F., Lum S.H., Zecca M., Porta F., Taskinen M.H., Gibson
B.E., Matthes-Martin S., Karakukcu M., Hauri-Hohl M.M., Veys P., Gennery A.R., Lucchini G., Felber M., Albert
M.H., Balashov D., Lankester A.C., Güngör T., Slatter M.
Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-
SCETIDE-PIDTC Study J Allergy Clin Immunol 2019
Ferrua F., Galimberti S., Courteille V., Slatter M.A., Booth C., Moshous D., Neven B., Blanche S., Cavazzana M.,
Laberko A., Shcherbina A., Balashov D., Soncini E., Porta F., Al-Mousa H., Al-Saud B., Al-Dhekri H., Arnaout R.,
Formankova R., Bertrand Y., Lange A., Smart J., Wolska-Kusnierz B., Aquino V.M., Dvorak C.C., Fasth A.,
Fouyssac F., Heilmann C., Hoenig M., Schuetz C., Kelecic J., Bredius R.G.M., Lankester A.C., Lindemans C.A.,
Suarez F., Sullivan K.E., Albert M.H., Kalwak K., Barlogis V., Bhatia M., Bordon V., Czogala W., Alonso L., Dogu F.,
Gozdzik J., Ikinciogullari A., Krivan G., Ljungman P., Meyts I., Mustillo P., Smith A.R., Speckmann C., Sundin M.,
Keogh S.J., Shaw P.J., Boelens J.J., Schulz A.S., Sedlacek P., Veys P., Mahlaoui N., Janda A., Davies E.G.,
Fischer A., Cowan M.J., Gennery A.R.
Hematopoietic Stem Cell Transplantation for Primary Immunodeficiencies Front Pediatr 2019
Gennery, A. R., Albert M. H., Slatter M. A., and Lankester A.
Hematopoietic Cell Transplantation for MHC Class II Deficiency Front Pediatr 2019
Lum, S. H., Neven B., Slatter M. A., and Gennery, A. R.
Conditioning perspectives for Primary Immunodeficiency Stem Cell Transplants Front Pediatr 2019
Shaw, P., Shizuru J., Hoenig M., and Veys P.
Hematopoietic Stem Cell Transplantation in Inborn Errors of Metabolism Front Pediatr 2019
Tan, E. Y., Boelens J. J., Jones S. A., and Wynn R. F.
Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Immune Dysregulatory Disorders Front
Pediatr 2019
Bakhtiar, S., Fekadu J., Seidel M. G., and Gambinieri E.
Wiskott-Aldrich Syndrome (WAS) and Dedicator of Cytokinesis 8- (DOCK8) Deficiency Front Pediatr 2019
Albert, M. H., & Freeman A. F.
The Evidence for Allogeneic Hematopoietic Stem Cell Transplantation for Congenital Neutrophil Disorders:
A Comprehensive Review by the Inborn Errors Working Party Group of the EBMT Front Pediatr 2019
Bakhtiar, S., Shadur, B., and Stepensky, P.
Long Term Outcome and Immune Function After Hematopoietic Stem Cell Transplantation for Primary
Immunodeficiency Front Pediatr 2019
Gennery, A. R., Lankester, A.
Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and
PDWP Working Parties Front Pediatr 2019
Greco R., Labopin M., Badoglio M., Veys P., Furtado Silva J.M., Abinun M., Gualandi F., Bornhauser M., Ciceri F.,
Saccardi R., Lankester A., Alexander T., Gennery A.R., Bader P., Farge D., Snowden J.A.
Universal Newborn Screening for Severe Combined Immunodeficiency (SCID) Front Pediatr 2019
van der Burg M., Mahlaoui N., Gaspar H.B., Pai S-Y.
Allogeneic HSCT in Adolescents and Young Adults With Primary Immunodeficiencies Front Pediatr 2019
Morris E.C., Albert M.H.
Haematopoietic Stem Cell Transplantation for Primary Haemophagocytic Lymphohistiocytosis Front
Pediatr 2019
Lehmberg K., Moshous D., Booth C.
Arjan Lankester (chair) Leiden, the Netherlands
Michael Albert (secretary/co-chair) Munich, Germany
Inborn Errors
Working Party
Arjan Lankester Chair [email protected]
Yvonne Wijkhuisen Secretary A. Lankester [email protected]
Michael Albert Secretary/Co-Chair [email protected]
Sheree Hazelaar EBMT Data manager [email protected]
Recent Publications on behalf of IEWP
StudiesEBMT Registry
Virtual IEWP events
Future Events 2020
Contact IEWP
Topic* Disease type Joint
AYA PID PID
Haplo in PID PID
ICF Syndrome PID
IFNg R1 R2 PID
Allo in MLD PID
Second Allo PID
Mevalonate Kinase
DeficiencyPID
CD27/CD70 deficiency PID IEWP – ESID
* If you whish to participate in data collection for these studies, please contact:
Tuesday September 1st
IEWP session
11.00 – 12.00
Auditorium 4
Program IEWP session:
▪ 11.00 – 11.12 Landscape of GT and allogeneic Therapy in IEM: The
next years - Robert Wynn
▪ 11.12 – 11.24 Therapeutic standards for patients with SCID- still a
matter of discussion - Manfred Hönig
▪ 11.24 – 11.36 Induction therapy in Inborn Errors of Immunity before
HSCT – Victoria Tesch
▪ 11.36 – 11.48 Inflammation and autoimmunity in PID: Does it matter for
HSCT? - Bénédicte Neven
▪ 11.48 – 12.00 Discussion
Significant Collaborations
