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Targeted busulfan-b...
Targeted busulfan-based reduced-intensity conditioning and HLA-matched HSCT cure hemophagocytic lymphohistiocytosis
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Felber, M. (author)
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Steward, C. G. (author)
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Kentouche, K. (author)
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- Fasth, Anders, 1945 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kliniska vetenskaper, Avdelningen för pediatrik,Institute of Clinical Sciences, Department of Pediatrics
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Wynn, R. F. (author)
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Zeilhofer, U. (author)
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Haunerdinger, V. (author)
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Volkmer, B. (author)
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Prader, S. (author)
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Gruhn, B. (author)
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Ehl, S. (author)
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Lehmberg, K. (author)
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Muller, D. (author)
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Gennery, A. R. (author)
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- Albert, M. H. (author)
- Karolinska Institutet
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Hauck, F. (author)
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Rao, K. (author)
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Veys, P. (author)
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Hassan, M. (author)
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Lankester, A. C. (author)
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Schmid, J. P. (author)
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Hauri-Hohl, M. M. (author)
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Gungor, T. (author)
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Inborn Errors Working Party, Iewp E. (author)
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European Soc Immunodeficiencies, E. S. (author)
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(creator_code:org_t)
- 2020-05-08
- 2020
- English.
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In: Blood Advances. - : American Society of Hematology. - 2473-9529 .- 2473-9537. ; 4:9, s. 1998-2010
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Abstract
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Publication and Content Type
- ref (subject category)
- art (subject category)
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- By the author/editor
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Felber, M.
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Steward, C. G.
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Kentouche, K.
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Fasth, Anders, 1 ...
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Wynn, R. F.
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Zeilhofer, U.
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show more...
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Haunerdinger, V.
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Volkmer, B.
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Prader, S.
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Gruhn, B.
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Ehl, S.
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Lehmberg, K.
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Muller, D.
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Gennery, A. R.
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Albert, M. H.
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Hauck, F.
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Rao, K.
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Veys, P.
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Hassan, M.
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Lankester, A. C.
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Schmid, J. P.
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Hauri-Hohl, M. M ...
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Gungor, T.
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Inborn Errors Wo ...
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European Soc Imm ...
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show less...
- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Hematology
- Articles in the publication
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Blood Advances
- By the university
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University of Gothenburg
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Karolinska Institutet