Skip to main content
Erschienen in: International Journal of Hematology 5/2017

09.02.2017 | Original Article

HLA haploidentical hematopoietic cell transplantation using clofarabine and busulfan for refractory pediatric hematological malignancy

verfasst von: Masatoshi Takagi, Yasuyoshi Ishiwata, Yuki Aoki, Satoshi Miyamoto, Akihiro Hoshino, Kazuaki Matsumoto, Akira Nishimura, Mari Tanaka, Masakatsu Yanagimachi, Noriko Mitsuiki, Kohsuke Imai, Hirokazu Kanegane, Michiko Kajiwara, Kanako Takikawa, Tsukasa Mae, Osamu Tomita, Junya Fujimura, Masato Yasuhara, Daisuke Tomizawa, Shuki Mizutani, Tomohiro Morio

Erschienen in: International Journal of Hematology | Ausgabe 5/2017

Einloggen, um Zugang zu erhalten

Abstract

Haploidentical hematopoietic cell transplantation (HCT) conditioning with clofarabine and target area under the blood concentration–time curve (AUC)-based busulfan adjustment was performed in three patients with refractory pediatric leukemia. The target AUC for two patients who had already received multiple transplantations was 3600 and 4000 μmol min/L, and that for the patient with Down’s syndrome was 3000 μmol min/L. Regimen-related toxicity was well tolerated in all cases. All three maintained cytological remission throughout the follow-up period (between 31 and 167 weeks). Thus, haploidentical HCT conditioning with clofarabine and target AUC-based busulfan adjustment may be a preferable option for children with recurrent or refractory pediatric leukemia.
Literatur
1.
Zurück zum Zitat Inagaki J, Fukano R, Noguchi M, Kurauchi K, Tanioka S, Okamura J. Hematopoietic stem cell transplantation following unsuccessful salvage treatment for relapsed acute lymphoblastic leukemia in children. Pediatr Blood Cancer. 2015;62:674–9. doi:10.1002/pbc.25353.CrossRefPubMed Inagaki J, Fukano R, Noguchi M, Kurauchi K, Tanioka S, Okamura J. Hematopoietic stem cell transplantation following unsuccessful salvage treatment for relapsed acute lymphoblastic leukemia in children. Pediatr Blood Cancer. 2015;62:674–9. doi:10.​1002/​pbc.​25353.CrossRefPubMed
2.
Zurück zum Zitat Poon LM, Bassett R Jr, Rondon G, Hamdi A, Qazilbash M, Hosing C, et al. Outcomes of second allogeneic hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia. Bone Marrow Transplant. 2013;48:666–70. doi:10.1038/bmt.2012.195.CrossRefPubMed Poon LM, Bassett R Jr, Rondon G, Hamdi A, Qazilbash M, Hosing C, et al. Outcomes of second allogeneic hematopoietic stem cell transplantation for patients with acute lymphoblastic leukemia. Bone Marrow Transplant. 2013;48:666–70. doi:10.​1038/​bmt.​2012.​195.CrossRefPubMed
4.
Zurück zum Zitat Kebriaei P, Basset R, Ledesma C, Ciurea S, Parmar S, Shpall EJ, et al. Clofarabine combined with busulfan provides excellent disease control in adult patients with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2012;18:1819–26. doi:10.1016/j.bbmt.2012.06.010.CrossRefPubMedPubMedCentral Kebriaei P, Basset R, Ledesma C, Ciurea S, Parmar S, Shpall EJ, et al. Clofarabine combined with busulfan provides excellent disease control in adult patients with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2012;18:1819–26. doi:10.​1016/​j.​bbmt.​2012.​06.​010.CrossRefPubMedPubMedCentral
5.
Zurück zum Zitat Magenau J, Tobai H, Pawarode A, Braun T, Peres E, Reddy P, et al. Clofarabine and busulfan conditioning facilitates engraftment and provides significant antitumor activity in nonremission hematologic malignancies. Blood. 2011;118:4258–64. doi:10.1182/blood-2011-06-358010.CrossRefPubMed Magenau J, Tobai H, Pawarode A, Braun T, Peres E, Reddy P, et al. Clofarabine and busulfan conditioning facilitates engraftment and provides significant antitumor activity in nonremission hematologic malignancies. Blood. 2011;118:4258–64. doi:10.​1182/​blood-2011-06-358010.CrossRefPubMed
6.
Zurück zum Zitat Watanabe E, Nishikawa T, Ikawa K, Yamaguchi H, Abematsu T, Nakagawa S, et al. Trough level monitoring of intravenous busulfan to estimate the area under the plasma drug concentration-time curve in pediatric hematopoietic stem cell transplant recipients. Int J Hematol. 2015;102:611–6. doi:10.1007/s12185-015-1853-6.CrossRefPubMed Watanabe E, Nishikawa T, Ikawa K, Yamaguchi H, Abematsu T, Nakagawa S, et al. Trough level monitoring of intravenous busulfan to estimate the area under the plasma drug concentration-time curve in pediatric hematopoietic stem cell transplant recipients. Int J Hematol. 2015;102:611–6. doi:10.​1007/​s12185-015-1853-6.CrossRefPubMed
7.
Zurück zum Zitat Aoki Y, Miyawaki R, Imai K, Takagi M, Kajiwara M, Ishiwata Y, et al. Haploidentical bone marrow transplantation with clofarabine and busulfan conditioning for a child with multiple recurrent acute lymphoblastic leukemia. J Pediatr Hematol Oncol. 2016;38:e39–41. doi:10.1097/MPH.0000000000000454.CrossRefPubMed Aoki Y, Miyawaki R, Imai K, Takagi M, Kajiwara M, Ishiwata Y, et al. Haploidentical bone marrow transplantation with clofarabine and busulfan conditioning for a child with multiple recurrent acute lymphoblastic leukemia. J Pediatr Hematol Oncol. 2016;38:e39–41. doi:10.​1097/​MPH.​0000000000000454​.CrossRefPubMed
8.
Zurück zum Zitat Bleyzac N, Barou P, Aulagner G. Rapid and sensitive high-performance liquid chromatographic method for busulfan assay in plasma. J Chromatogr B Biomed Sci Appl. 2000;742:427–32.CrossRefPubMed Bleyzac N, Barou P, Aulagner G. Rapid and sensitive high-performance liquid chromatographic method for busulfan assay in plasma. J Chromatogr B Biomed Sci Appl. 2000;742:427–32.CrossRefPubMed
10.
Zurück zum Zitat Hijiya N, Thomson B, Isakoff MS, Silverman LB, Steinherz PG, Borowitz MJ, et al. Phase 2 trial of clofarabine in combination with etoposide and cyclophosphamide in pediatric patients with refractory or relapsed acute lymphoblastic leukemia. Blood. 2011;118:6043–9. doi:10.1182/blood-2011-08-374710.CrossRefPubMedPubMedCentral Hijiya N, Thomson B, Isakoff MS, Silverman LB, Steinherz PG, Borowitz MJ, et al. Phase 2 trial of clofarabine in combination with etoposide and cyclophosphamide in pediatric patients with refractory or relapsed acute lymphoblastic leukemia. Blood. 2011;118:6043–9. doi:10.​1182/​blood-2011-08-374710.CrossRefPubMedPubMedCentral
11.
Zurück zum Zitat Russell JA, Tran HT, Quinlan D, Chaudhry A, Duggan P, Brown C, et al. Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes. Biol Blood Marrow Transplant. 2002;8:468–76.CrossRefPubMed Russell JA, Tran HT, Quinlan D, Chaudhry A, Duggan P, Brown C, et al. Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes. Biol Blood Marrow Transplant. 2002;8:468–76.CrossRefPubMed
12.
Zurück zum Zitat Ryu SG, Lee JH, Choi SJ, Lee JH, Lee YS, Seol M, et al. Randomized comparison of four-times-daily versus once-daily intravenous busulfan in conditioning therapy for hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2007;13:1095–105. doi:10.1016/j.bbmt.2007.06.005.CrossRefPubMed Ryu SG, Lee JH, Choi SJ, Lee JH, Lee YS, Seol M, et al. Randomized comparison of four-times-daily versus once-daily intravenous busulfan in conditioning therapy for hematopoietic cell transplantation. Biol Blood Marrow Transplant. 2007;13:1095–105. doi:10.​1016/​j.​bbmt.​2007.​06.​005.CrossRefPubMed
13.
Zurück zum Zitat Ward J, Kletzel M, Duerst R, Fuleihan R, Chaudhury S, Schneiderman J, et al. Single Daily Busulfan Dosing for Infants with Nonmalignant Diseases Undergoing Reduced-Intensity Conditioning for Allogeneic Hematopoietic Progenitor Cell Transplantation. Biol Blood Marrow Transplant. 2015;21:1612–21. doi:10.1016/j.bbmt.2015.05.017.CrossRefPubMed Ward J, Kletzel M, Duerst R, Fuleihan R, Chaudhury S, Schneiderman J, et al. Single Daily Busulfan Dosing for Infants with Nonmalignant Diseases Undergoing Reduced-Intensity Conditioning for Allogeneic Hematopoietic Progenitor Cell Transplantation. Biol Blood Marrow Transplant. 2015;21:1612–21. doi:10.​1016/​j.​bbmt.​2015.​05.​017.CrossRefPubMed
14.
Zurück zum Zitat Bartelink IH, Bredius RG, Ververs TT, Raphael MF, van Kesteren C, Bierings M, et al. Once-daily intravenous busulfan with therapeutic drug monitoring compared to conventional oral busulfan improves survival and engraftment in children undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant. 2008;14:88–98. doi:10.1016/j.bbmt.2007.09.015.CrossRefPubMed Bartelink IH, Bredius RG, Ververs TT, Raphael MF, van Kesteren C, Bierings M, et al. Once-daily intravenous busulfan with therapeutic drug monitoring compared to conventional oral busulfan improves survival and engraftment in children undergoing allogeneic stem cell transplantation. Biol Blood Marrow Transplant. 2008;14:88–98. doi:10.​1016/​j.​bbmt.​2007.​09.​015.CrossRefPubMed
15.
Zurück zum Zitat Gonzalez-Vicent M, Molina B, Perez A, Diaz MA. Once-daily intravenous busulfan for 47 pediatric patients undergoing autologous hematopoietic stem cell transplantation: a single center study. J Pediatr Hematol Oncol. 2012;34:180–3. doi:10.1097/MPH.0b013e3182431e1b.CrossRefPubMed Gonzalez-Vicent M, Molina B, Perez A, Diaz MA. Once-daily intravenous busulfan for 47 pediatric patients undergoing autologous hematopoietic stem cell transplantation: a single center study. J Pediatr Hematol Oncol. 2012;34:180–3. doi:10.​1097/​MPH.​0b013e3182431e1b​.CrossRefPubMed
16.
Zurück zum Zitat Pasquini MC, Le-Rademacher J, Zhu X, Artz A, DiPersio J, Fernandez HF, et al. Intravenous busulfan-based myeloablative conditioning regimens prior to hematopoietic cell transplantation for hematologic malignancies. Biol Blood Marrow Transplant. 2016;22:1424–30. doi:10.1016/j.bbmt.2016.04.013.CrossRefPubMed Pasquini MC, Le-Rademacher J, Zhu X, Artz A, DiPersio J, Fernandez HF, et al. Intravenous busulfan-based myeloablative conditioning regimens prior to hematopoietic cell transplantation for hematologic malignancies. Biol Blood Marrow Transplant. 2016;22:1424–30. doi:10.​1016/​j.​bbmt.​2016.​04.​013.CrossRefPubMed
17.
Zurück zum Zitat Magenau J, Westervelt P, Khaled S, McGuirk J, Hari P, Eapen M, et al. A multicenter trial of myeloablative clofarabine and busulfan conditioning for relapsed or primary induction failure AML not in remission at the time of allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2016;. doi:10.1038/bmt.2016.188.PubMedCentral Magenau J, Westervelt P, Khaled S, McGuirk J, Hari P, Eapen M, et al. A multicenter trial of myeloablative clofarabine and busulfan conditioning for relapsed or primary induction failure AML not in remission at the time of allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2016;. doi:10.​1038/​bmt.​2016.​188.PubMedCentral
Metadaten
Titel
HLA haploidentical hematopoietic cell transplantation using clofarabine and busulfan for refractory pediatric hematological malignancy
verfasst von
Masatoshi Takagi
Yasuyoshi Ishiwata
Yuki Aoki
Satoshi Miyamoto
Akihiro Hoshino
Kazuaki Matsumoto
Akira Nishimura
Mari Tanaka
Masakatsu Yanagimachi
Noriko Mitsuiki
Kohsuke Imai
Hirokazu Kanegane
Michiko Kajiwara
Kanako Takikawa
Tsukasa Mae
Osamu Tomita
Junya Fujimura
Masato Yasuhara
Daisuke Tomizawa
Shuki Mizutani
Tomohiro Morio
Publikationsdatum
09.02.2017
Verlag
Springer Japan
Erschienen in
International Journal of Hematology / Ausgabe 5/2017
Print ISSN: 0925-5710
Elektronische ISSN: 1865-3774
DOI
https://doi.org/10.1007/s12185-017-2187-3

Weitere Artikel der Ausgabe 5/2017

International Journal of Hematology 5/2017 Zur Ausgabe

Adjuvante Immuntherapie verlängert Leben bei RCC

25.04.2024 Nierenkarzinom Nachrichten

Nun gibt es auch Resultate zum Gesamtüberleben: Eine adjuvante Pembrolizumab-Therapie konnte in einer Phase-3-Studie das Leben von Menschen mit Nierenzellkarzinom deutlich verlängern. Die Sterberate war im Vergleich zu Placebo um 38% geringer.

Alectinib verbessert krankheitsfreies Überleben bei ALK-positivem NSCLC

25.04.2024 NSCLC Nachrichten

Das Risiko für Rezidiv oder Tod von Patienten und Patientinnen mit reseziertem ALK-positivem NSCLC ist unter einer adjuvanten Therapie mit dem Tyrosinkinase-Inhibitor Alectinib signifikant geringer als unter platinbasierter Chemotherapie.

Bei Senioren mit Prostatakarzinom auf Anämie achten!

24.04.2024 DGIM 2024 Nachrichten

Patienten, die zur Behandlung ihres Prostatakarzinoms eine Androgendeprivationstherapie erhalten, entwickeln nicht selten eine Anämie. Wer ältere Patienten internistisch mitbetreut, sollte auf diese Nebenwirkung achten.

ICI-Therapie in der Schwangerschaft wird gut toleriert

Müssen sich Schwangere einer Krebstherapie unterziehen, rufen Immuncheckpointinhibitoren offenbar nicht mehr unerwünschte Wirkungen hervor als andere Mittel gegen Krebs.

Update Onkologie

Bestellen Sie unseren Fach-Newsletter und bleiben Sie gut informiert.