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Items: 1 to 20 of 103

1.

Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency.

Aydin SE, Freeman AF, Al-Herz W, Al-Mousa HA, Arnaout RK, Aydin RC, Barlogis V, Belohradsky BH, Bonfim C, Bredius RG, Chu JI, Ciocarlie OC, Doğu F, Gaspar HB, Geha RS, Gennery AR, Hauck F, Hawwari A, Hickstein DD, Hoenig M, Ikinciogullari A, Klein C, Kumar A, Ifversen MRS, Matthes S, Metin A, Neven B, Pai SY, Parikh SH, Picard C, Renner ED, Sanal Ö, Schulz AS, Schuster F, Shah NN, Shereck EB, Slatter MA, Su HC, van Montfrans J, Woessmann W, Ziegler JB, Albert MH; Inborn Errors Working Party of the European Group for Blood and Marrow Transplantation and the European Society for Primary Immunodeficiencies.

J Allergy Clin Immunol Pract. 2019 Mar;7(3):848-855. doi: 10.1016/j.jaip.2018.10.035. Epub 2018 Nov 2.

PMID:
30391550
2.

Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency.

Al-Herz W, Chu JI, van der Spek J, Raghupathy R, Massaad MJ, Keles S, Biggs CM, Cockerton L, Chou J, Dbaibo G, Elisofon SA, Hanna-Wakim R, Kim HB, Lehmann LE, McDonald DR, Notarangelo LD, Veys P, Chatila TA, Geha RS, Gaspar HB, Pai SY.

J Allergy Clin Immunol. 2016 Sep;138(3):852-859.e3. doi: 10.1016/j.jaci.2016.02.022. Epub 2016 Apr 6.

3.

Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience.

Morillo-Gutierrez B, Beier R, Rao K, Burroughs L, Schulz A, Ewins AM, Gibson B, Sedlacek P, Krol L, Strahm B, Zaidman I, Kalwak K, Talano JA, Woolfrey A, Fraser C, Meyts I, Müller I, Wachowiak J, Bernardo ME, Veys P, Sykora KW, Gennery AR, Slatter M.

Blood. 2016 Jul 21;128(3):440-8. doi: 10.1182/blood-2016-03-704015. Epub 2016 May 23. Erratum in: Blood. 2016 Nov 24;128(21):2585.

4.

Clinical and immunological correction of DOCK8 deficiency by allogeneic hematopoietic stem cell transplantation following a reduced toxicity conditioning regimen.

Boztug H, Karitnig-Weiß C, Ausserer B, Renner ED, Albert MH, Sawalle-Belohradsky J, Belohradsky BH, Mann G, Horcher E, Rümmele-Waibel A, Geyeregger R, Lakatos K, Peters C, Lawitschka A, Matthes-Martin S.

Pediatr Hematol Oncol. 2012 Oct;29(7):585-94. Epub 2012 Aug 16.

PMID:
22897717
5.

Matched related and unrelated donor hematopoietic stem cell transplantation for DOCK8 deficiency.

Cuellar-Rodriguez J, Freeman AF, Grossman J, Su H, Parta M, Murdock H, Shah N, Bollard C, Kong HH, Moutsopoulos N, Stone K, Gea-Banacloche J, Holland SM, Hickstein DD.

Biol Blood Marrow Transplant. 2015 Jun;21(6):1037-45. doi: 10.1016/j.bbmt.2015.01.022. Epub 2015 Jan 27.

6.

Outcome of treosulfan-based reduced-toxicity conditioning regimens for HSCT in high-risk patients with primary immune deficiencies.

Haskoloğlu Ş, Köstel Bal S, İslamoğlu C, Altun D, Kendirli T, Doğu EF, İkincioğulları A.

Pediatr Transplant. 2018 Nov;22(7):e13266. doi: 10.1111/petr.13266. Epub 2018 Jul 11.

PMID:
29992714
7.

Clofarabine Plus Busulfan is an Effective Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Acute Lymphoblastic Leukemia: Long-Term Study Results.

Kebriaei P, Bassett R, Lyons G, Valdez B, Ledesma C, Rondon G, Oran B, Ciurea S, Alousi A, Popat U, Patel K, Ahmed S, Olson A, Bashir Q, Shah N, Jones R, Marin D, Rezvani K, Nieto Y, Khouri I, Qazilbash M, Hosing C, Shpall E, Champlin RE, Andersson BS.

Biol Blood Marrow Transplant. 2017 Feb;23(2):285-292. doi: 10.1016/j.bbmt.2016.11.001. Epub 2016 Nov 2.

8.

Hematopoietic stem cell transplant effectively rescues lymphocyte differentiation and function in DOCK8-deficient patients.

Pillay BA, Avery DT, Smart JM, Cole T, Choo S, Chan D, Gray PE, Frith K, Mitchell R, Phan TG, Wong M, Campbell DE, Hsu P, Ziegler JB, Peake J, Alvaro F, Picard C, Bustamante J, Neven B, Cant AJ, Uzel G, Arkwright PD, Casanova JL, Su HC, Freeman AF, Shah N, Hickstein DD, Tangye SG, Ma CS.

JCI Insight. 2019 Apr 25;5. pii: 127527. doi: 10.1172/jci.insight.127527.

9.

Treosulfan-based conditioning regimen for allogeneic haematopoietic stem cell transplantation in children with sickle cell disease.

Strocchio L, Zecca M, Comoli P, Mina T, Giorgiani G, Giraldi E, Vinti L, Merli P, Regazzi M, Locatelli F.

Br J Haematol. 2015 Jun;169(5):726-36. doi: 10.1111/bjh.13352. Epub 2015 Mar 27.

PMID:
25818248
10.

Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Chronic and Advanced Phase Myelofibrosis.

Keyzner A, Han S, Shapiro S, Moshier E, Schorr E, Petersen B, Najfeld V, Kremyanskaya M, Isola L, Hoffman R, Mascarenhas J.

Biol Blood Marrow Transplant. 2016 Dec;22(12):2180-2186. doi: 10.1016/j.bbmt.2016.08.029. Epub 2016 Sep 3.

11.

Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study.

Güngör T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, Vermont C, Ahmad I, Shaw PJ, Telles da Cunha JM, Schlegel PG, Hough R, Fasth A, Kentouche K, Gruhn B, Fernandes JF, Lachance S, Bredius R, Resnick IB, Belohradsky BH, Gennery A, Fischer A, Gaspar HB, Schanz U, Seger R, Rentsch K, Veys P, Haddad E, Albert MH, Hassan M; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation.

Lancet. 2014 Feb 1;383(9915):436-48. doi: 10.1016/S0140-6736(13)62069-3. Epub 2013 Oct 23.

PMID:
24161820
12.

A Bortezomib-Based Regimen Offers Promising Survival and Graft-versus-Host Disease Prophylaxis in Myeloablative HLA-Mismatched and Unrelated Donor Transplantation: A Phase II Trial.

Koreth J, Kim HT, Lange PB, Bindra B, Reynolds CG, Chammas MJ, Armand P, Cutler CS, Ho VT, Glotzbecker B, Nikiforow S, Ritz J, Blazar BR, Soiffer RJ, Antin JH, Alyea EP 3rd.

Biol Blood Marrow Transplant. 2015 Nov;21(11):1907-13. doi: 10.1016/j.bbmt.2015.05.027. Epub 2015 Jun 6.

14.

Tandem Orthotopic Living Donor Liver Transplantation Followed by Same Donor Haploidentical Hematopoietic Stem Cell Transplantation for DOCK8 Deficiency: Tandem Liver and Bone Marrow Transplant in DOCK8 Deficiency.

Freeman AF, Yazigi N, Shah NN, Kleiner DE, Parta M, Atkinson P, Heller T, Holland SM, Kaufman SS, Khan KM, Hickstein DD.

Transplantation. 2019 Jan 30. doi: 10.1097/TP.0000000000002649. [Epub ahead of print]

PMID:
30720689
15.

Survival Advantage and Comparable Toxicity in Reduced-Toxicity Treosulfan-Based versus Reduced-Intensity Busulfan-Based Conditioning Regimen in Myelodysplastic Syndrome and Acute Myeloid Leukemia Patients after Allogeneic Hematopoietic Cell Transplantation.

Sakellari I, Mallouri D, Gavriilaki E, Batsis I, Kaliou M, Constantinou V, Papalexandri A, Lalayanni C, Vadikolia C, Athanasiadou A, Yannaki E, Sotiropoulos D, Smias C, Anagnostopoulos A.

Biol Blood Marrow Transplant. 2017 Mar;23(3):445-451. doi: 10.1016/j.bbmt.2016.11.023. Epub 2016 Nov 30.

16.

Busulfan dose intensity and outcomes in reduced-intensity allogeneic peripheral blood stem cell transplantation for myelodysplastic syndrome or acute myeloid leukemia.

Chen YB, Coughlin E, Kennedy KF, Alyea EP, Armand P, Attar EC, Ballen KK, Cutler C, Dey BR, Koreth J, McAfee SL, Spitzer TR, Antin JH, Soiffer RJ, Ho VT.

Biol Blood Marrow Transplant. 2013 Jun;19(6):981-7. doi: 10.1016/j.bbmt.2013.03.016. Epub 2013 Apr 2.

17.

Survival after second hematopoietic stem cell transplantation for recurrent pediatric acute myeloid leukemia.

Meshinchi S, Leisenring WM, Carpenter PA, Woolfrey AE, Sievers EL, Radich JP, Sanders JE.

Biol Blood Marrow Transplant. 2003 Nov;9(11):706-13.

18.

Fludarabine-based conditioning secures engraftment of second hematopoietic stem cell allografts (HSCT) in the treatment of initial graft failure.

Chewning JH, Castro-Malaspina H, Jakubowski A, Kernan NA, Papadopoulos EB, Small TN, Heller G, Hsu KC, Perales MA, van den Brink MR, Young JW, Prockop SE, Collins NH, O'Reilly RJ, Boulad F.

Biol Blood Marrow Transplant. 2007 Nov;13(11):1313-23. Epub 2007 Sep 7.

19.

Post-transplantation Cyclophosphamide and Sirolimus after Haploidentical Hematopoietic Stem Cell Transplantation Using a Treosulfan-based Myeloablative Conditioning and Peripheral Blood Stem Cells.

Cieri N, Greco R, Crucitti L, Morelli M, Giglio F, Levati G, Assanelli A, Carrabba MG, Bellio L, Milani R, Lorentino F, Stanghellini MT, De Freitas T, Marktel S, Bernardi M, Corti C, Vago L, Bonini C, Ciceri F, Peccatori J.

Biol Blood Marrow Transplant. 2015 Aug;21(8):1506-14. doi: 10.1016/j.bbmt.2015.04.025. Epub 2015 May 19.

20.

Successful long-term correction of autosomal recessive hyper-IgE syndrome due to DOCK8 deficiency by hematopoietic stem cell transplantation.

Bittner TC, Pannicke U, Renner ED, Notheis G, Hoffmann F, Belohradsky BH, Wintergerst U, Hauser M, Klein B, Schwarz K, Schmid I, Albert MH.

Klin Padiatr. 2010 Nov;222(6):351-5. doi: 10.1055/s-0030-1265135. Epub 2010 Nov 5.

PMID:
21058221

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