Skip to main content
Top
Published in: Annals of Hematology 3/2021

01-03-2021 | Graft-Versus-Host Disease | Original Article

A multicenter phase II study of intrabone single-unit cord blood transplantation without antithymocyte globulin

Authors: Tetsuya Nishida, Takeshi Kobayashi, Masashi Sawa, Shinichi Masuda, Yasuhiko Shibasaki, Tatsunori Goto, Noriko Fukuhara, Nobuharu Fujii, Kazuhiro Ikegame, Junichi Sugita, Takashi Ikeda, Yachiyo Kuwatsuka, Ritsuro Suzuki, Yuho Najima, Noriko Doki, Tomonori Kato, Yuichiro Inagaki, Yoshikazu Utsu, Nobuyuki Aotsuka, Masayoshi Masuko, Seitaro Terakura, Yasushi Onishi, Yoshinobu Maeda, Masaya Okada, Takanori Teshima, Makoto Murata

Published in: Annals of Hematology | Issue 3/2021

Login to get access

Abstract

To overcome the delayed or failed engraftment after unrelated cord blood transplantation (CBT), we conducted a multicenter phase II study of intrabone single-unit CBT without antithymocyte globulin (ATG) for adult patients with hematological malignancies (UMIN-CTR, UMIN000020997). Sixty-four patients received an intrabone injection of unwashed (n = 61) or washed (n = 3) cord blood after local anesthesia. All injection-related adverse events were mild and resolved spontaneously. Sixty-two patients were evaluable for the efficacy of intrabone CBT of serological HLA-A, -B, and -DR ≥ 4/6 matched cord blood with a median number of 2.57 × 107/kg cryopreserved total nucleated cells. The probability of survival with neutrophil engraftment on day 28 was 77.4% (95% confidence interval, 67.0–85.8%), which exceeded the threshold value. The cumulative incidences of neutrophils ≥ 0.5 × 109/L on day 60 was 80.6% (68.2–88.6%), with a median time to recovery of 21 days after transplantation. The cumulative incidences of platelets ≥ 20 × 109/L and platelets ≥ 50 × 109/L on day 100 were 75.8% (62.6–84.9%) and 72.6% (59.4–82.1%), respectively, with median time to platelets ≥ 20 × 109/L and platelets ≥ 50 × 109/L of 38 and 45 days after transplantation, respectively. The cumulative incidences of grade II–IV and III–IV acute graft-versus-host disease were 29.0% and 6.5%, respectively. All responded to steroid therapy, and secondary treatments were not required. The present study suggests the efficacy of intrabone single-unit CBT without ATG in terms of early engraftment and controllable acute graft-versus-host disease.
Literature
2.
go back to reference Esquirol A, Querol S, Garcia-Cadenas I, Novelli S, Garrido A, Saavedra S et al (2020) When an HLA identical donor is not available in adults with hematological neoplasms: single-center comparison of single-unit cord blood transplantation and haploidentical-related PBSC transplantation with PTCy using a standardized conditioning platform (thiotepa-busulfan-fludarabine). Ann Hematol 99:157–165PubMedCrossRef Esquirol A, Querol S, Garcia-Cadenas I, Novelli S, Garrido A, Saavedra S et al (2020) When an HLA identical donor is not available in adults with hematological neoplasms: single-center comparison of single-unit cord blood transplantation and haploidentical-related PBSC transplantation with PTCy using a standardized conditioning platform (thiotepa-busulfan-fludarabine). Ann Hematol 99:157–165PubMedCrossRef
3.
go back to reference Eapen M, Rocha V, Sanz G, Scaradavou A, Zhang MJ, Arcese W, Sirvent A, Champlin RE, Chao N, Gee AP, Isola L, Laughlin MJ, Marks DI, Nabhan S, Ruggeri A, Soiffer R, Horowitz MM, Gluckman E, Wagner JE (2010) Effect of graft source on unrelated donor haemopoietic stem-cell transplantation in adults with acute leukaemia: a retrospective analysis. Lancet Oncol. 11:653–660PubMedPubMedCentralCrossRef Eapen M, Rocha V, Sanz G, Scaradavou A, Zhang MJ, Arcese W, Sirvent A, Champlin RE, Chao N, Gee AP, Isola L, Laughlin MJ, Marks DI, Nabhan S, Ruggeri A, Soiffer R, Horowitz MM, Gluckman E, Wagner JE (2010) Effect of graft source on unrelated donor haemopoietic stem-cell transplantation in adults with acute leukaemia: a retrospective analysis. Lancet Oncol. 11:653–660PubMedPubMedCentralCrossRef
4.
go back to reference Terakura S, Nishida T, Sawa M, Kato T, Miyao K, Ozawa Y et al (2020) Prospective evaluation of alternative donor from unrelated donor and cord blood in adult acute leukemia and myelodysplastic syndrome. Bone Marrow Transplant 55:1399–1409PubMedCrossRef Terakura S, Nishida T, Sawa M, Kato T, Miyao K, Ozawa Y et al (2020) Prospective evaluation of alternative donor from unrelated donor and cord blood in adult acute leukemia and myelodysplastic syndrome. Bone Marrow Transplant 55:1399–1409PubMedCrossRef
5.
go back to reference Kanda J, Ichinohe T, Kato S, Uchida N, Terakura S, Fukuda T et al (2013) Unrelated cord blood transplantation vs related transplantation with HLA 1-antigen mismatch in the graft-versus-host direction. Leukemia. 27:286–294PubMedCrossRef Kanda J, Ichinohe T, Kato S, Uchida N, Terakura S, Fukuda T et al (2013) Unrelated cord blood transplantation vs related transplantation with HLA 1-antigen mismatch in the graft-versus-host direction. Leukemia. 27:286–294PubMedCrossRef
6.
go back to reference Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, McGlave PB, Miller JS et al (2005) Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood. 105:1343–1347PubMedCrossRef Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, McGlave PB, Miller JS et al (2005) Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood. 105:1343–1347PubMedCrossRef
7.
go back to reference Scaradavou A, Brunstein CG, Eapen M, Le-Rademacher J, Barker JN, Chao N et al (2013) Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia. Blood. 121:752–758PubMedPubMedCentralCrossRef Scaradavou A, Brunstein CG, Eapen M, Le-Rademacher J, Barker JN, Chao N et al (2013) Double unit grafts successfully extend the application of umbilical cord blood transplantation in adults with acute leukemia. Blood. 121:752–758PubMedPubMedCentralCrossRef
8.
go back to reference Kai S, Wake A, Okada M, Kurata M, Atsuta Y, Ishikawa J, Nakamae H, Aotsuka N, Kasai M, Misawa M, Taniguchi S, Kato S (2013) Double-unit cord blood transplantation after myeloablative conditioning for patients with hematologic malignancies: a multicenter phase II study in Japan. Biol Blood Marrow Transplant. 19:812–819PubMedCrossRef Kai S, Wake A, Okada M, Kurata M, Atsuta Y, Ishikawa J, Nakamae H, Aotsuka N, Kasai M, Misawa M, Taniguchi S, Kato S (2013) Double-unit cord blood transplantation after myeloablative conditioning for patients with hematologic malignancies: a multicenter phase II study in Japan. Biol Blood Marrow Transplant. 19:812–819PubMedCrossRef
9.
go back to reference Wagner JE Jr, Eapen M, Carter S, Wang Y, Schultz KR, Wall DA, Bunin N, Delaney C, Haut P, Margolis D, Peres E, Verneris MR, Walters M, Horowitz MM, Kurtzberg J (2014) One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med 371:1685–1694PubMedPubMedCentralCrossRef Wagner JE Jr, Eapen M, Carter S, Wang Y, Schultz KR, Wall DA, Bunin N, Delaney C, Haut P, Margolis D, Peres E, Verneris MR, Walters M, Horowitz MM, Kurtzberg J (2014) One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med 371:1685–1694PubMedPubMedCentralCrossRef
10.
go back to reference Michel G, Galambrun C, Sirvent A, Pochon C, Bruno B, Jubert C, Loundou A, Yakoub-Agha I, Milpied N, Lutz P, Marie-Cardine A, Gandemer V, Blaise D, Michallet M, Rialland F, Renard C, Oudin C, Esmiol S, Seux M, Baumstarck K, Mohty M, Rocha V, Dalle JH (2016) Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome. Blood. 127:3450–3457PubMedCrossRef Michel G, Galambrun C, Sirvent A, Pochon C, Bruno B, Jubert C, Loundou A, Yakoub-Agha I, Milpied N, Lutz P, Marie-Cardine A, Gandemer V, Blaise D, Michallet M, Rialland F, Renard C, Oudin C, Esmiol S, Seux M, Baumstarck K, Mohty M, Rocha V, Dalle JH (2016) Single- vs double-unit cord blood transplantation for children and young adults with acute leukemia or myelodysplastic syndrome. Blood. 127:3450–3457PubMedCrossRef
11.
go back to reference Delaney C, Heimfeld S, Brashem-Stein C, Voorhies H, Manger RL, Bernstein ID (2010) Notch-mediated expansion of human cord blood progenitor cells capable of rapid myeloid reconstitution. Nat Med 16:232–236PubMedPubMedCentralCrossRef Delaney C, Heimfeld S, Brashem-Stein C, Voorhies H, Manger RL, Bernstein ID (2010) Notch-mediated expansion of human cord blood progenitor cells capable of rapid myeloid reconstitution. Nat Med 16:232–236PubMedPubMedCentralCrossRef
12.
go back to reference de Lima M, McNiece I, Robinson SN, Munsell M, Eapen M, Horowitz M, Alousi A, Saliba R, McMannis JD, Kaur I, Kebriaei P, Parmar S, Popat U, Hosing C, Champlin R, Bollard C, Molldrem JJ, Jones RB, Nieto Y, Andersson BS, Shah N, Oran B, Cooper LJN, Worth L, Qazilbash MH, Korbling M, Rondon G, Ciurea S, Bosque D, Maewal I, Simmons PJ, Shpall EJ (2012) Cord-blood engraftment with ex vivo mesenchymal-cell coculture. N Engl J Med 367:2305–2315PubMedPubMedCentralCrossRef de Lima M, McNiece I, Robinson SN, Munsell M, Eapen M, Horowitz M, Alousi A, Saliba R, McMannis JD, Kaur I, Kebriaei P, Parmar S, Popat U, Hosing C, Champlin R, Bollard C, Molldrem JJ, Jones RB, Nieto Y, Andersson BS, Shah N, Oran B, Cooper LJN, Worth L, Qazilbash MH, Korbling M, Rondon G, Ciurea S, Bosque D, Maewal I, Simmons PJ, Shpall EJ (2012) Cord-blood engraftment with ex vivo mesenchymal-cell coculture. N Engl J Med 367:2305–2315PubMedPubMedCentralCrossRef
13.
go back to reference Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G (2019) Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol 37:367–374PubMedCrossRef Horwitz ME, Wease S, Blackwell B, Valcarcel D, Frassoni F, Boelens JJ, Nierkens S, Jagasia M, Wagner JE, Kuball J, Koh LP, Majhail NS, Stiff PJ, Hanna R, Hwang WYK, Kurtzberg J, Cilloni D, Freedman LS, Montesinos P, Sanz G (2019) Phase I/II study of stem-cell transplantation using a single cord blood unit expanded ex vivo with nicotinamide. J Clin Oncol 37:367–374PubMedCrossRef
14.
go back to reference Frassoni F, Gualandi F, Podestà M, Raiola AM, Ibatici A, Piaggio G, Sessarego M, Sessarego N, Gobbi M, Sacchi N, Labopin M, Bacigalupo A (2008) Direct intrabone transplant of unrelated cord-blood cells in acute leukaemia: a phase I/II study. Lancet Oncol 9:831–839PubMedCrossRef Frassoni F, Gualandi F, Podestà M, Raiola AM, Ibatici A, Piaggio G, Sessarego M, Sessarego N, Gobbi M, Sacchi N, Labopin M, Bacigalupo A (2008) Direct intrabone transplant of unrelated cord-blood cells in acute leukaemia: a phase I/II study. Lancet Oncol 9:831–839PubMedCrossRef
15.
go back to reference Murata M, Maeda Y, Masuko M, Onishi Y, Endo T, Terakura S, Ishikawa Y, Iriyama C, Ushijima Y, Goto T, Fujii N, Tanimoto M, Kobayashi H, Shibasaki Y, Fukuhara N, Inamoto Y, Suzuki R, Kodera Y, Matsushita T, Kiyoi H, Naoe T, Nishida T (2017) Phase II study of intrabone single unit cord blood transplantation for hematological malignancies. Cancer Sci 108:1634–1639PubMedPubMedCentralCrossRef Murata M, Maeda Y, Masuko M, Onishi Y, Endo T, Terakura S, Ishikawa Y, Iriyama C, Ushijima Y, Goto T, Fujii N, Tanimoto M, Kobayashi H, Shibasaki Y, Fukuhara N, Inamoto Y, Suzuki R, Kodera Y, Matsushita T, Kiyoi H, Naoe T, Nishida T (2017) Phase II study of intrabone single unit cord blood transplantation for hematological malignancies. Cancer Sci 108:1634–1639PubMedPubMedCentralCrossRef
16.
go back to reference Kurita N, Gosho M, Yokoyama Y, Kato T, Obara N, Sakata-Yanagimoto M, Hasegawa Y, Uchida N, Takahashi S, Kouzai Y, Atsuta Y, Kurata M, Ichinohe T, Chiba S (2017) A phase I/II trial of intrabone marrow cord blood transplantation and comparison of the hematological recovery with the Japanese nationwide database. Bone Marrow Transplant 52:574–579PubMedCrossRef Kurita N, Gosho M, Yokoyama Y, Kato T, Obara N, Sakata-Yanagimoto M, Hasegawa Y, Uchida N, Takahashi S, Kouzai Y, Atsuta Y, Kurata M, Ichinohe T, Chiba S (2017) A phase I/II trial of intrabone marrow cord blood transplantation and comparison of the hematological recovery with the Japanese nationwide database. Bone Marrow Transplant 52:574–579PubMedCrossRef
17.
go back to reference Okada M, Tasaka T, Ikegame K, Aotsuka N, Kobayashi T, Najima Y, Matsuhashi Y, Wada H, Tokunaga H, Masuda S, Utsu Y, Yoshihara S, Kaida K, Daimon T, Ogawa H (2018) A prospective multicenter phase II study of intrabone marrow transplantation of unwashed cord blood using reduced-intensity conditioning. Eur J Haematol 100:335–343PubMedCrossRef Okada M, Tasaka T, Ikegame K, Aotsuka N, Kobayashi T, Najima Y, Matsuhashi Y, Wada H, Tokunaga H, Masuda S, Utsu Y, Yoshihara S, Kaida K, Daimon T, Ogawa H (2018) A prospective multicenter phase II study of intrabone marrow transplantation of unwashed cord blood using reduced-intensity conditioning. Eur J Haematol 100:335–343PubMedCrossRef
18.
go back to reference Bonifazi F, Dan E, Labopin M, Sessa M, Guadagnuolo V, Ferioli M, Rizzi S, de Carolis S, Sinigaglia B, Motta MR, Bontadini A, Giudice V, Martinelli G, Arpinati M, Cavo M, Bonafé M, Storci G (2019) Intrabone transplant provides full stemness of cord blood stem cells with fast hematopoietic recovery and low GVHD rate: results from a prospective study. Bone Marrow Transplant 54:717–725PubMedCrossRef Bonifazi F, Dan E, Labopin M, Sessa M, Guadagnuolo V, Ferioli M, Rizzi S, de Carolis S, Sinigaglia B, Motta MR, Bontadini A, Giudice V, Martinelli G, Arpinati M, Cavo M, Bonafé M, Storci G (2019) Intrabone transplant provides full stemness of cord blood stem cells with fast hematopoietic recovery and low GVHD rate: results from a prospective study. Bone Marrow Transplant 54:717–725PubMedCrossRef
19.
go back to reference Davies SM, Kollman C, Anasetti C, Antin JH, Gajewski J, Casper JT, Nademanee A, Noreen H, King R, Confer D, Kernan NA (2000) Engraftment and survival after unrelated-donor bone marrow transplantation: a report from the national marrow donor program. Blood. 96:4096–4102PubMedCrossRef Davies SM, Kollman C, Anasetti C, Antin JH, Gajewski J, Casper JT, Nademanee A, Noreen H, King R, Confer D, Kernan NA (2000) Engraftment and survival after unrelated-donor bone marrow transplantation: a report from the national marrow donor program. Blood. 96:4096–4102PubMedCrossRef
20.
go back to reference Morishima Y, Sasazuki T, Inoko H, Juji T, Akaza T, Yamamoto K et al (2002) The clinical significance of human leukocyte antigen (HLA) allele compatibility in patients receiving a marrow transplant from serologically HLA-A, HLA-B, and HLA-DR matched unrelated donors. Blood. 99:4200–4206PubMedCrossRef Morishima Y, Sasazuki T, Inoko H, Juji T, Akaza T, Yamamoto K et al (2002) The clinical significance of human leukocyte antigen (HLA) allele compatibility in patients receiving a marrow transplant from serologically HLA-A, HLA-B, and HLA-DR matched unrelated donors. Blood. 99:4200–4206PubMedCrossRef
21.
go back to reference Anasetti C, Logan BR, Lee SJ, Waller EK, Weisdorf DJ, Wingard JR, Cutler CS, Westervelt P, Woolfrey A, Couban S, Ehninger G, Johnston L, Maziarz RT, Pulsipher MA, Porter DL, Mineishi S, McCarty JM, Khan SP, Anderlini P, Bensinger WI, Leitman SF, Rowley SD, Bredeson C, Carter SL, Horowitz MM, Confer DL (2012) Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med 367:1487–1496PubMedCrossRef Anasetti C, Logan BR, Lee SJ, Waller EK, Weisdorf DJ, Wingard JR, Cutler CS, Westervelt P, Woolfrey A, Couban S, Ehninger G, Johnston L, Maziarz RT, Pulsipher MA, Porter DL, Mineishi S, McCarty JM, Khan SP, Anderlini P, Bensinger WI, Leitman SF, Rowley SD, Bredeson C, Carter SL, Horowitz MM, Confer DL (2012) Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med 367:1487–1496PubMedCrossRef
22.
go back to reference Rubinstein P, Dobrila L, Rosenfield RE, Adamson JW, Migliaccio G, Migliaccio AR, Taylor PE, Stevens CE (1995) Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci U S A 92:10119–10122PubMedPubMedCentralCrossRef Rubinstein P, Dobrila L, Rosenfield RE, Adamson JW, Migliaccio G, Migliaccio AR, Taylor PE, Stevens CE (1995) Processing and cryopreservation of placental/umbilical cord blood for unrelated bone marrow reconstitution. Proc Natl Acad Sci U S A 92:10119–10122PubMedPubMedCentralCrossRef
23.
go back to reference Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J et al (1995) 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant 15:825–828PubMed Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatty P, Hows J et al (1995) 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant 15:825–828PubMed
24.
go back to reference Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ, Martin P, Chien J, Przepiorka D, Couriel D, Cowen EW, Dinndorf P, Farrell A, Hartzman R, Henslee-Downey J, Jacobsohn D, McDonald G, Mittleman B, Rizzo JD, Robinson M, Schubert M, Schultz K, Shulman H, Turner M, Vogelsang G, Flowers MED (2005) National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report Biol Blood Marrow Transplant 11:945–956CrossRef Filipovich AH, Weisdorf D, Pavletic S, Socie G, Wingard JR, Lee SJ, Martin P, Chien J, Przepiorka D, Couriel D, Cowen EW, Dinndorf P, Farrell A, Hartzman R, Henslee-Downey J, Jacobsohn D, McDonald G, Mittleman B, Rizzo JD, Robinson M, Schubert M, Schultz K, Shulman H, Turner M, Vogelsang G, Flowers MED (2005) National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report Biol Blood Marrow Transplant 11:945–956CrossRef
25.
go back to reference Giralt S, Ballen K, Rizzo D, Bacigalupo A, Horowitz M, Pasquini M, Sandmaier B (2009) Reduced-intensity conditioning regimen workshop: defining the dose spectrum. Report of a workshop convened by the center for international blood and marrow transplant research Biol Blood Marrow Transplant 15:367–369CrossRef Giralt S, Ballen K, Rizzo D, Bacigalupo A, Horowitz M, Pasquini M, Sandmaier B (2009) Reduced-intensity conditioning regimen workshop: defining the dose spectrum. Report of a workshop convened by the center for international blood and marrow transplant research Biol Blood Marrow Transplant 15:367–369CrossRef
26.
go back to reference Kanda Y (2013) Investigation of the freely available easy-to-use software 'EZR' for medical statistics. Bone Marrow Transplant 48:452–458PubMedCrossRef Kanda Y (2013) Investigation of the freely available easy-to-use software 'EZR' for medical statistics. Bone Marrow Transplant 48:452–458PubMedCrossRef
27.
go back to reference Fuji S, Oshima K, Ohashi K, Sawa M, Saito T, Eto T, Tanaka M, Onizuka M, Nakamae H, Shiratori S, Ozawa Y, Hidaka M, Nagamura-Inoue T, Tanaka H, Fukuda T, Ichinohe T, Atsuta Y, Ogata M (2020) Impact of pretransplant donor-specific anti-HLA antibodies on cord blood transplantation on behalf of the Transplant Complications Working Group of Japan Society for Hematopoietic Cell Transplantation. Bone Marrow Transplant 55:722–728PubMedCrossRef Fuji S, Oshima K, Ohashi K, Sawa M, Saito T, Eto T, Tanaka M, Onizuka M, Nakamae H, Shiratori S, Ozawa Y, Hidaka M, Nagamura-Inoue T, Tanaka H, Fukuda T, Ichinohe T, Atsuta Y, Ogata M (2020) Impact of pretransplant donor-specific anti-HLA antibodies on cord blood transplantation on behalf of the Transplant Complications Working Group of Japan Society for Hematopoietic Cell Transplantation. Bone Marrow Transplant 55:722–728PubMedCrossRef
28.
go back to reference Kanda J, Hayashi H, Ruggeri A, Kimura F, Volt F, Takahashi S, Labopin M, Kako S, Tozatto-Maio K, Yano S, Sanz G, Uchida N, van Lint MT, Kato S, Mohty M, Forcade E, Kanamori H, Sierra J, Ohno Y, Saccardi R, Fukuda T, Ichinohe T, Takanashi M, Rocha V, Okamoto S, Nagler A, Atsuta Y, Gluckman E (2020) Prognostic factors for adult single cord blood transplantation among European and Japanese populations: the Eurocord/ALWP-EBMT and JSHCT/JDCHCT collaborative study. Leukemia. 34:128–137PubMedCrossRef Kanda J, Hayashi H, Ruggeri A, Kimura F, Volt F, Takahashi S, Labopin M, Kako S, Tozatto-Maio K, Yano S, Sanz G, Uchida N, van Lint MT, Kato S, Mohty M, Forcade E, Kanamori H, Sierra J, Ohno Y, Saccardi R, Fukuda T, Ichinohe T, Takanashi M, Rocha V, Okamoto S, Nagler A, Atsuta Y, Gluckman E (2020) Prognostic factors for adult single cord blood transplantation among European and Japanese populations: the Eurocord/ALWP-EBMT and JSHCT/JDCHCT collaborative study. Leukemia. 34:128–137PubMedCrossRef
29.
go back to reference Rubinstein P, Carrier C, Scaradavou A, Kurtzberg J, Adamson J, Migliaccio AR, Berkowitz RL, Cabbad M, Dobrila NL, Taylor PE, Rosenfield RE, Stevens CE (1998) Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339:1565–1577PubMedCrossRef Rubinstein P, Carrier C, Scaradavou A, Kurtzberg J, Adamson J, Migliaccio AR, Berkowitz RL, Cabbad M, Dobrila NL, Taylor PE, Rosenfield RE, Stevens CE (1998) Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339:1565–1577PubMedCrossRef
30.
go back to reference Wagner JE, Barker JN, DeFor TE, Baker KS, Blazar BR, Eide C et al (2002) Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood. 100:1611–1618PubMedCrossRef Wagner JE, Barker JN, DeFor TE, Baker KS, Blazar BR, Eide C et al (2002) Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood. 100:1611–1618PubMedCrossRef
31.
go back to reference Dehn J, Spellman S, Hurley CK, Shaw BE, Barker JN, Burns LJ, Confer DL, Eapen M, Fernandez-Vina M, Hartzman R, Maiers M, Marino SR, Mueller C, Perales MA, Rajalingam R, Pidala J (2019) Selection of unrelated donors and cord blood units for hematopoietic cell transplantation: guidelines from the NMDP/CIBMTR. Blood. 134:924–934PubMedPubMedCentralCrossRef Dehn J, Spellman S, Hurley CK, Shaw BE, Barker JN, Burns LJ, Confer DL, Eapen M, Fernandez-Vina M, Hartzman R, Maiers M, Marino SR, Mueller C, Perales MA, Rajalingam R, Pidala J (2019) Selection of unrelated donors and cord blood units for hematopoietic cell transplantation: guidelines from the NMDP/CIBMTR. Blood. 134:924–934PubMedPubMedCentralCrossRef
32.
go back to reference Terakura S, Azuma E, Murata M, Kumamoto T, Hirayama M, Atsuta Y, Kodera Y, Yazaki M, Naoe T, Kato K (2007) Hematopoietic engraftment in recipients of unrelated donor umbilical cord blood is affected by the CD34+ and CD8+ cell doses. Biol Blood Marrow Transplant 13:822–830PubMedCrossRef Terakura S, Azuma E, Murata M, Kumamoto T, Hirayama M, Atsuta Y, Kodera Y, Yazaki M, Naoe T, Kato K (2007) Hematopoietic engraftment in recipients of unrelated donor umbilical cord blood is affected by the CD34+ and CD8+ cell doses. Biol Blood Marrow Transplant 13:822–830PubMedCrossRef
33.
go back to reference Konuma T, Kato S, Oiwa-Monna M, Tanoue S, Ogawa M, Isobe M et al (2017) Cryopreserved CD34(+) cell dose, but not total nucleated cell dose. Influences Hematopoietic Recovery and Extensive Chronic Graft-versus-Host Disease after Single-Unit Cord Blood Transplantation in Adult Patients Biol Blood Marrow Transplant 23:1142–1150PubMed Konuma T, Kato S, Oiwa-Monna M, Tanoue S, Ogawa M, Isobe M et al (2017) Cryopreserved CD34(+) cell dose, but not total nucleated cell dose. Influences Hematopoietic Recovery and Extensive Chronic Graft-versus-Host Disease after Single-Unit Cord Blood Transplantation in Adult Patients Biol Blood Marrow Transplant 23:1142–1150PubMed
34.
go back to reference Nakasone H, Tabuchi K, Uchida N, Ohno Y, Matsuhashi Y, Takahashi S, Onishi Y, Onizuka M, Kobayashi H, Fukuda T, Ichinohe T, Takanashi M, Kato K, Atsuta Y, Yabe H, Kanda Y (2019) Which is more important for the selection of cord blood units for haematopoietic cell transplantation: the number of CD34-positive cells or total nucleated cells? Br J Haematol 185:166–169PubMedCrossRef Nakasone H, Tabuchi K, Uchida N, Ohno Y, Matsuhashi Y, Takahashi S, Onishi Y, Onizuka M, Kobayashi H, Fukuda T, Ichinohe T, Takanashi M, Kato K, Atsuta Y, Yabe H, Kanda Y (2019) Which is more important for the selection of cord blood units for haematopoietic cell transplantation: the number of CD34-positive cells or total nucleated cells? Br J Haematol 185:166–169PubMedCrossRef
35.
go back to reference Kurtzberg J, Prasad VK, Carter SL, Wagner JE, Baxter-Lowe LA, Wall D, Kapoor N, Guinan EC, Feig SA, Wagner EL, Kernan NA (2008) Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies. Blood. 112:4318–4327PubMedPubMedCentralCrossRef Kurtzberg J, Prasad VK, Carter SL, Wagner JE, Baxter-Lowe LA, Wall D, Kapoor N, Guinan EC, Feig SA, Wagner EL, Kernan NA (2008) Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies. Blood. 112:4318–4327PubMedPubMedCentralCrossRef
36.
go back to reference Barker JN, Scaradavou A, Stevens CE (2010) Combined effect of total nucleated cell dose and HLA match on transplantation outcome in 1061 cord blood recipients with hematologic malignancies. Blood. 115:1843–1849PubMedPubMedCentralCrossRef Barker JN, Scaradavou A, Stevens CE (2010) Combined effect of total nucleated cell dose and HLA match on transplantation outcome in 1061 cord blood recipients with hematologic malignancies. Blood. 115:1843–1849PubMedPubMedCentralCrossRef
37.
go back to reference Eapen M, Klein JP, Ruggeri A, Spellman S, Lee SJ, Anasetti C, Arcese W, Barker JN, Baxter-Lowe LA, Brown M, Fernandez-Vina MA, Freeman J, He W, Iori AP, Horowitz MM, Locatelli F, Marino S, Maiers M, Michel G, Sanz GF, Gluckman E, Rocha V (2014) Impact of allele-level HLA matching on outcomes after myeloablative single unit umbilical cord blood transplantation for hematologic malignancy. Blood. 123:133–140PubMedPubMedCentralCrossRef Eapen M, Klein JP, Ruggeri A, Spellman S, Lee SJ, Anasetti C, Arcese W, Barker JN, Baxter-Lowe LA, Brown M, Fernandez-Vina MA, Freeman J, He W, Iori AP, Horowitz MM, Locatelli F, Marino S, Maiers M, Michel G, Sanz GF, Gluckman E, Rocha V (2014) Impact of allele-level HLA matching on outcomes after myeloablative single unit umbilical cord blood transplantation for hematologic malignancy. Blood. 123:133–140PubMedPubMedCentralCrossRef
38.
go back to reference Yokoyama H, Morishima Y, Fuji S, Uchida N, Takahashi S, Onizuka M, Tanaka M, Yuju O, Eto T, Ozawa Y, Takada S, Takanashi M, Kato K, Kanda Y, Ichinohe T, Atsuta Y, Kanda J (2020) Impact of HLA allele mismatch at HLA-A, -B, -C, and -DRB1 in single cord blood transplantation. Biol Blood Marrow Transplant. 26:519–528PubMedCrossRef Yokoyama H, Morishima Y, Fuji S, Uchida N, Takahashi S, Onizuka M, Tanaka M, Yuju O, Eto T, Ozawa Y, Takada S, Takanashi M, Kato K, Kanda Y, Ichinohe T, Atsuta Y, Kanda J (2020) Impact of HLA allele mismatch at HLA-A, -B, -C, and -DRB1 in single cord blood transplantation. Biol Blood Marrow Transplant. 26:519–528PubMedCrossRef
39.
go back to reference Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, Miller JS, Wagner JE (2003) Rapid and complete donor chimerism in adult recipients of unrelated donor umbilical cord blood transplantation after reduced-intensity conditioning. Blood. 102:1915–1919PubMedCrossRef Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, Miller JS, Wagner JE (2003) Rapid and complete donor chimerism in adult recipients of unrelated donor umbilical cord blood transplantation after reduced-intensity conditioning. Blood. 102:1915–1919PubMedCrossRef
40.
go back to reference Chen YB, Wang T, Hemmer MT, Brady C, Couriel DR, Alousi A, Pidala J, Urbano-Ispizua A, Choi SW, Nishihori T, Teshima T, Inamoto Y, Wirk B, Marks DI, Abdel-Azim H, Lehmann L, Yu L, Bitan M, Cairo MS, Qayed M, Salit R, Gale RP, Martino R, Jaglowski S, Bajel A, Savani B, Frangoul H, Lewis ID, Storek J, Askar M, Kharfan-Dabaja MA, Aljurf M, Ringden O, Reshef R, Olsson RF, Hashmi S, Seo S, Spitzer TR, MacMillan ML, Lazaryan A, Spellman SR, Arora M, Cutler CS (2017) GvHD after umbilical cord blood transplantation for acute leukemia: an analysis of risk factors and effect on outcomes. Bone Marrow Transplant 52:400–408PubMedCrossRef Chen YB, Wang T, Hemmer MT, Brady C, Couriel DR, Alousi A, Pidala J, Urbano-Ispizua A, Choi SW, Nishihori T, Teshima T, Inamoto Y, Wirk B, Marks DI, Abdel-Azim H, Lehmann L, Yu L, Bitan M, Cairo MS, Qayed M, Salit R, Gale RP, Martino R, Jaglowski S, Bajel A, Savani B, Frangoul H, Lewis ID, Storek J, Askar M, Kharfan-Dabaja MA, Aljurf M, Ringden O, Reshef R, Olsson RF, Hashmi S, Seo S, Spitzer TR, MacMillan ML, Lazaryan A, Spellman SR, Arora M, Cutler CS (2017) GvHD after umbilical cord blood transplantation for acute leukemia: an analysis of risk factors and effect on outcomes. Bone Marrow Transplant 52:400–408PubMedCrossRef
41.
go back to reference Bonifazi F, Rubio MT, Bacigalupo A, Boelens JJ, Finke J, Greinix H et al (2020) Rabbit ATG/ATLG in preventing graft-versus-host disease after allogeneic stem cell transplantation: consensus-based recommendations by an international expert panel. Bone Marrow Transplant 55:1093–1102PubMedPubMedCentralCrossRef Bonifazi F, Rubio MT, Bacigalupo A, Boelens JJ, Finke J, Greinix H et al (2020) Rabbit ATG/ATLG in preventing graft-versus-host disease after allogeneic stem cell transplantation: consensus-based recommendations by an international expert panel. Bone Marrow Transplant 55:1093–1102PubMedPubMedCentralCrossRef
42.
go back to reference Laughlin MJ, Barker J, Bambach B, Koc ON, Rizzieri DA, Wagner JE, Gerson SL, Lazarus HM, Cairo M, Stevens CE, Rubinstein P, Kurtzberg J (2001) Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J Med 344:1815–1822PubMedCrossRef Laughlin MJ, Barker J, Bambach B, Koc ON, Rizzieri DA, Wagner JE, Gerson SL, Lazarus HM, Cairo M, Stevens CE, Rubinstein P, Kurtzberg J (2001) Hematopoietic engraftment and survival in adult recipients of umbilical-cord blood from unrelated donors. N Engl J Med 344:1815–1822PubMedCrossRef
43.
go back to reference Rocha V, Labopin M, Sanz G, Arcese W, Schwerdtfeger R, Bosi A, Jacobsen N, Ruutu T, de Lima M, Finke J, Frassoni F, Gluckman E (2004) Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia. N Engl J Med 351:2276–2285PubMedCrossRef Rocha V, Labopin M, Sanz G, Arcese W, Schwerdtfeger R, Bosi A, Jacobsen N, Ruutu T, de Lima M, Finke J, Frassoni F, Gluckman E (2004) Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia. N Engl J Med 351:2276–2285PubMedCrossRef
44.
go back to reference Hiwarkar P, Hubank M, Qasim W, Chiesa R, Gilmour KC, Saudemont A, Amrolia PJ, Veys P (2017) Cord blood transplantation recapitulates fetal ontogeny with a distinct molecular signature that supports CD4(+) T-cell reconstitution. Blood Adv. 1:2206–2216PubMedPubMedCentralCrossRef Hiwarkar P, Hubank M, Qasim W, Chiesa R, Gilmour KC, Saudemont A, Amrolia PJ, Veys P (2017) Cord blood transplantation recapitulates fetal ontogeny with a distinct molecular signature that supports CD4(+) T-cell reconstitution. Blood Adv. 1:2206–2216PubMedPubMedCentralCrossRef
45.
go back to reference Politikos I, Lavery JA, Hilden P, Cho C, Borrill T, Maloy MA, Giralt SA, van den Brink MRM, Perales MA, Barker JN (2020) Robust CD4+ T-cell recovery in adults transplanted with cord blood and no antithymocyte globulin. Blood Adv 4:191–202PubMedPubMedCentralCrossRef Politikos I, Lavery JA, Hilden P, Cho C, Borrill T, Maloy MA, Giralt SA, van den Brink MRM, Perales MA, Barker JN (2020) Robust CD4+ T-cell recovery in adults transplanted with cord blood and no antithymocyte globulin. Blood Adv 4:191–202PubMedPubMedCentralCrossRef
46.
go back to reference Wakamatsu M, Terakura S, Ohashi K, Fukuda T, Ozawa Y, Kanamori H, Sawa M, Uchida N, Ota S, Matsushita A, Kanda Y, Nakamae H, Ichinohe T, Kato K, Murata M, Atsuta Y, Teshima T (2019) Impacts of thymoglobulin in patients with acute leukemia in remission undergoing allogeneic HSCT from different donors. Blood Adv. 3:105–115PubMedPubMedCentralCrossRef Wakamatsu M, Terakura S, Ohashi K, Fukuda T, Ozawa Y, Kanamori H, Sawa M, Uchida N, Ota S, Matsushita A, Kanda Y, Nakamae H, Ichinohe T, Kato K, Murata M, Atsuta Y, Teshima T (2019) Impacts of thymoglobulin in patients with acute leukemia in remission undergoing allogeneic HSCT from different donors. Blood Adv. 3:105–115PubMedPubMedCentralCrossRef
47.
go back to reference Rocha V, Labopin M, Ruggeri A, Podestà M, Gallamini A, Bonifazi F, Sanchez-Guijo FM, Rovira M, Socie G, Baltadakis I, Michallet M, Deconinck E, Bacigalupo A, Mohty M, Gluckman E, Frassoni F (2013) Unrelated cord blood transplantation: outcomes after single-unit intrabone injection compared with double-unit intravenous injection in patients with hematological malignancies. Transplantation. 95:1284–1291PubMedCrossRef Rocha V, Labopin M, Ruggeri A, Podestà M, Gallamini A, Bonifazi F, Sanchez-Guijo FM, Rovira M, Socie G, Baltadakis I, Michallet M, Deconinck E, Bacigalupo A, Mohty M, Gluckman E, Frassoni F (2013) Unrelated cord blood transplantation: outcomes after single-unit intrabone injection compared with double-unit intravenous injection in patients with hematological malignancies. Transplantation. 95:1284–1291PubMedCrossRef
48.
go back to reference Hägglund H, Ringdén O, Agren B, Wennberg L, Remberger M, Rundquist L et al (1998) Intraosseous compared to intravenous infusion of allogeneic bone marrow. Bone Marrow Transplant 21:331–335PubMedCrossRef Hägglund H, Ringdén O, Agren B, Wennberg L, Remberger M, Rundquist L et al (1998) Intraosseous compared to intravenous infusion of allogeneic bone marrow. Bone Marrow Transplant 21:331–335PubMedCrossRef
49.
go back to reference Hägglund H, Remberger M, Ringden O (2014) Twenty-year follow-up of a randomized trial comparing intraosseous and i.v. BM transplantation. Bone Marrow Transplant 49:1541–1542PubMedCrossRef Hägglund H, Remberger M, Ringden O (2014) Twenty-year follow-up of a randomized trial comparing intraosseous and i.v. BM transplantation. Bone Marrow Transplant 49:1541–1542PubMedCrossRef
Metadata
Title
A multicenter phase II study of intrabone single-unit cord blood transplantation without antithymocyte globulin
Authors
Tetsuya Nishida
Takeshi Kobayashi
Masashi Sawa
Shinichi Masuda
Yasuhiko Shibasaki
Tatsunori Goto
Noriko Fukuhara
Nobuharu Fujii
Kazuhiro Ikegame
Junichi Sugita
Takashi Ikeda
Yachiyo Kuwatsuka
Ritsuro Suzuki
Yuho Najima
Noriko Doki
Tomonori Kato
Yuichiro Inagaki
Yoshikazu Utsu
Nobuyuki Aotsuka
Masayoshi Masuko
Seitaro Terakura
Yasushi Onishi
Yoshinobu Maeda
Masaya Okada
Takanori Teshima
Makoto Murata
Publication date
01-03-2021
Publisher
Springer Berlin Heidelberg
Published in
Annals of Hematology / Issue 3/2021
Print ISSN: 0939-5555
Electronic ISSN: 1432-0584
DOI
https://doi.org/10.1007/s00277-020-04365-z

Other articles of this Issue 3/2021

Annals of Hematology 3/2021 Go to the issue