WFRF:(Steward C. G.)
Sökning: WFRF:(Steward C. G.) > Targeted busulfan-b...
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| Fältnamn | Indikatorer | Metadata |
|---|---|---|
| 000 | 04504naa a2200613 4500 | |
| 001 | oai:gup.ub.gu.se/294250 | |
| 003 | SwePub | |
| 008 | 240528s2020 | |||||||||||000 ||eng| | |
| 009 | oai:prod.swepub.kib.ki.se:143685111 | |
| 024 | 7 | a https://gup.ub.gu.se/publication/2942502 URI |
| 024 | 7 | a https://doi.org/10.1182/bloodadvances.20200017482 DOI |
| 024 | 7 | a http://kipublications.ki.se/Default.aspx?queryparsed=id:1436851112 URI |
| 040 | a (SwePub)gud (SwePub)ki | |
| 041 | a eng | |
| 042 | 9 SwePub | |
| 072 | 7 | a ref2 swepub-contenttype |
| 072 | 7 | a art2 swepub-publicationtype |
| 100 | 1 | a Felber, M.4 aut |
| 245 | 1 0 | a Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis |
| 264 | c 2020-05-08 | |
| 264 | 1 | b American Society of Hematology,c 2020 |
| 520 | a Reduced-intensity/reduced-toxicity conditioning and allogeneic T-cell replete hematopoietic stem cell transplantation are curative in patients with hemophagocytic lymphohistiocytosis (HLH). Unstable donor chimerism (DC) and relapses are clinical challenges. We examined the effect of a reduced-intensity conditioning regimen based on targeted busulfan to enhance myeloid DC in HLH. The European Society for Bone and Marrow Transplantation-approved reduced-intensity conditioning protocol comprised targeted submyeloablative IV busulfan, IV fludarabine, and serotherapy comprising IV alemtuzumab (0.5-0.8 mg/kg) for unrelated-donor and IV rabbit anti-T-cell globulin for related-donor transplants. We assessed toxicity, engraftment, graft-versus-host disease (GHVD), DC in blood cell subtypes, and overall survival/event-free survival. Twenty-five patients from 7 centers were treated (median age, 0.68 year). The median total dose and cumulative area under the curve of busulfan was 13.1 mg/kg (6.4-26.4) and 63.1 mg/L x h (48-77), respectively. Bone marrow, peripheral blood stem cell, or cord blood transplants from HLA-matched related (n = 7) or unrelated (n = 18) donors were administered. Donor cells engrafted in all patients (median: neutrophils d+20/platelets d128). At last follow-up (median, 36 months; range, 8-111 months), the median DC of CD151 neutrophils, CD3(+) T cells, and CD16(+)56(+) natural killer cells was 99.5% (10-100), 97% (30-100), and 97.5% (30-100), respectively. Eight patients (32%) developed sinusoidal obstruction syndrome, resolving after defibrotide treatment. The 3-year overall survival and event-free survival rates were both 100%. None of the patients developed acute grade III to IV GHVD. Limited chronic GVHD was encountered in 4%. This regimen achieves excellent results with stable DC in patients with HLH. | |
| 650 | 7 | a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Hematologi0 (SwePub)302022 hsv//swe |
| 650 | 7 | a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Hematology0 (SwePub)302022 hsv//eng |
| 700 | 1 | a Steward, C. G.4 aut |
| 700 | 1 | a Kentouche, K.4 aut |
| 700 | 1 | a Fasth, Anders,d 1945u Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics4 aut0 (Swepub:gu)xfasan |
| 700 | 1 | a Wynn, R. F.4 aut |
| 700 | 1 | a Zeilhofer, U.4 aut |
| 700 | 1 | a Haunerdinger, V.4 aut |
| 700 | 1 | a Volkmer, B.4 aut |
| 700 | 1 | a Prader, S.4 aut |
| 700 | 1 | a Gruhn, B.4 aut |
| 700 | 1 | a Ehl, S.4 aut |
| 700 | 1 | a Lehmberg, K.4 aut |
| 700 | 1 | a Muller, D.4 aut |
| 700 | 1 | a Gennery, A. R.4 aut |
| 700 | 1 | a Albert, M. H.u Karolinska Institutet4 aut |
| 700 | 1 | a Hauck, F.4 aut |
| 700 | 1 | a Rao, K.4 aut |
| 700 | 1 | a Veys, P.4 aut |
| 700 | 1 | a Hassan, M.4 aut |
| 700 | 1 | a Lankester, A. C.4 aut |
| 700 | 1 | a Schmid, J. P.4 aut |
| 700 | 1 | a Hauri-Hohl, M. M.4 aut |
| 700 | 1 | a Gungor, T.4 aut |
| 700 | 1 | a Inborn Errors Working Party, Iewp E.4 aut |
| 700 | 1 | a European Soc Immunodeficiencies, E. S.4 aut |
| 710 | 2 | a Göteborgs universitetb Institutionen för kliniska vetenskaper, Avdelningen för pediatrik4 org |
| 773 | 0 | t Blood Advancesd : American Society of Hematologyg 4:9, s. 1998-2010q 4:9<1998-2010x 2473-9529x 2473-9537 |
| 856 | 4 | u https://ashpublications.org/bloodadvances/article-pdf/4/9/1998/1755516/advancesadv2020001748.pdf |
| 856 | 4 8 | u https://gup.ub.gu.se/publication/294250 |
| 856 | 4 8 | u https://doi.org/10.1182/bloodadvances.2020001748 |
| 856 | 4 8 | u http://kipublications.ki.se/Default.aspx?queryparsed=id:143685111 |
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