Reduced intensity HLA-haploidentical BMT with post transplantation cyclophosphamide in nonmalignant hematologic diseases

被引：110

作者：

Brodsky, R. A. ^{[1
,2
]}

Luznik, L. ^{[2
]}

Bolanos-Meade, J. ^{[2
]}

Leffell, M. S. ^{[3
]}

Jones, R. J. ^{[1
,2
]}

Fuchs, E. J. ^{[2
]}

机构：

[1] Johns Hopkins Univ, Sch Med, Div Hematol, Baltimore, MD 21205 USA

[2] Johns Hopkins Univ, Sch Med, Dept Oncol, Sidney Kimmel Comprehens Canc Ctr, Baltimore, MD 21205 USA

[3] Johns Hopkins Univ, Sch Med, Div Immunogenet, Baltimore, MD 21205 USA

来源：

BONE MARROW TRANSPLANTATION | 2008年 / 42卷 / 08期

关键词：

paroxysmal nocturnal hemoglobinuria; sickle cell anemia; cyclophosphamide; haploidentical;

D O I：

10.1038/bmt.2008.203

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

Allogeneic blood or marrow transplantation (BMT) is potentially curative for a variety of life-threatening nonmalignant hematologic diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and hemoglobinopathies. The application of BMT to treat these disorders is limited by the lack of suitable donors and often end-organ damage from the underlying disease. We treated three patients with thrombotic PNH, one of whom also had sickle cell disease, with a nonmyeloablative, HLA-haploidentical BMT with post-transplant CY. Rapid engraftment without GVHD occurred in two of the patients, including the patient with sickle cell disease. Both patients are disease free with full donor chimerism and require no immunosuppressive therapy, with follow-up of 1 and 4 years, respectively. Nonmyeloablative, HLA-haploidentical BMT with post-transplant CY is a promising approach for patients with life-threatening nonmalignant hematologic disease who lack an HLA-matched sibling donor.

引用

页码：523 / 527

页数：5

共 26 条

[1] CLUES TO THE PATHOGENESIS OF FAMILIAL COLORECTAL-CANCER [J].

AALTONEN, LA ;

PELTOMAKI, P ;

LEACH, FS ;

SISTONEN, P ;

PYLKKANEN, L ;

MECKLIN, JP ;

JARVINEN, H ;

POWELL, SM ;

JEN, J ;

HAMILTON, SR ;

PETERSEN, GM ;

KINZLER, KW ;

VOGELSTEIN, B ;

DELACHAPELLE, A .

SCIENCE, 1993, 260 (5109) :812-816

[2] Unrelated cord blood transplantation in children with sickle cell disease: Review of four-center experience [J].

Adamkiewicz, Tom V. ;

Szabolcs, Paul ;

Haight, Ann ;

Baker, K. Scott ;

Staba, Susan ;

Kedar, Amos ;

Chiang, K. Y. ;

Krishnamurti, Lakshmanan ;

Boyer, Michael W. ;

Kurtzberg, Joan ;

Wagner, John E. ;

Wingard, John R. ;

Yeager, Andrew M. .

PEDIATRIC TRANSPLANTATION, 2007, 11 (06) :641-644

[3] Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease [J].

Bernaudin, Francoise ;

Socie, Gerard ;

Kuentz, Mathieu ;

Chevret, Sylvie ;

Duval, Michel ;

Bertrand, Yves ;

Vannier, Jean-Pierre ;

Yakouben, Karima ;

Thuret, Isabelle ;

Bordigoni, Pierre ;

Fischer, Alain ;

Lutz, Patrick ;

Stephan, Jean-Louis ;

Dhedin, Nathalie ;

Plouvier, Emmanuel ;

Margueritte, Genevieve ;

Bories, Dominique ;

Verlhac, Suzanne ;

Esperou, Helene ;

Coic, Lena ;

Vernant, Jean-Paul ;

Gluckman, Eliane .

BLOOD, 2007, 110 (07) :2749-2756

[4] Complete remission in severe aplastic anemia after high-dose cyclophosphamide without bone marrow transplantation [J].

Brodsky, RA ;

Sensenbrenner, LL ;

Jones, RJ .

BLOOD, 1996, 87 (02) :491-494

[5] Durable treatment-free remission after high-dose cyclophosphamide therapy for previously untreated severe aplastic anemia [J].

Brodsky, RA ;

Sensenbrenner, LL ;

Smith, BD ;

Dorr, D ;

Seaman, PJ ;

Lee, SM ;

Karp, JE ;

Brodsky, I ;

Jones, RJ .

ANNALS OF INTERNAL MEDICINE, 2001, 135 (07) :477-483

[6] Elimination of alloantibodies by immunoablative high dose cyclophosphamide [J].

Brodsky, RA ;

Fuller, AK ;

Ratner, LE ;

Leffell, MS ;

Jones, RJ .

TRANSPLANTATION, 2001, 71 (03) :482-484

[7] Immunoablative high-dose cyclophosphamide without stem-cell rescue for refractory, severe autoimmune disease [J].

Brodsky, RA ;

Petri, M ;

Smith, BD ;

Seifter, EJ ;

Spivak, JL ;

Styler, M ;

Dang, CV ;

Brodsky, I ;

Jones, RJ .

ANNALS OF INTERNAL MEDICINE, 1998, 129 (12) :1031-1035

[8] Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria [J].

Brodsky, Robert A. ;

Young, Neal S. ;

Antonioli, Elisabetta ;

Risitano, Antonio M. ;

Schrezenmeier, Hubert ;

Schubert, Jorg ;

Gaya, Anna ;

Coyle, Luke ;

De Castro, Carlos ;

Fu, Chieh-Lin ;

Maciejewski, Jaroslaw P. ;

Bessler, Monica ;

Kroon, Henk-Andre ;

Rother, Russell P. ;

Hillmen, Peter .

BLOOD, 2008, 111 (04) :1840-1847

[9] Interphase FISH for Y chromosome, VNTR polymorphisms, and RT-PCR for BCR-ABL in the monitoring of HLA-matched and mismatched transplants [J].

Crescenzi, B ;

Fizzotti, M ;

Piattoni, S ;

La Starza, R ;

Matteucci, C ;

Carotti, A ;

Aversa, F ;

Martelli, MF ;

Mecucci, C .

CANCER GENETICS AND CYTOGENETICS, 2000, 120 (01) :25-29

[10] The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria [J].

Hillmen, Peter ;

Young, Neal S. ;

Schubert, Joerg ;

Brodsky, Robert A. ;

Socie, Gerard ;

Muus, Petra ;

Roeth, Alexander ;

Szer, Jeffrey ;

Elebute, Modupe O. ;

Nakamura, Ryotaro ;

Browne, Paul ;

Risitano, Antonio M. ;

Hill, Anita ;

Schrezenmeier, Hubert ;

Fu, Chieh-Lin ;

Maciejewski, Jaroslaw ;

Rollins, Scott A. ;

Mojcik, Christopher F. ;

Rother, Russell P. ;

Luzzatto, Lucio .

NEW ENGLAND JOURNAL OF MEDICINE, 2006, 355 (12) :1233-1243

← 1 2 3 →