Allogeneic Hematopoietic Stem Cell Transplantation for Adults with Sickle Cell Disease

被引：9

作者：

Saraf, Santosh L. ^{[1
]}

Rondelli, Damiano ^{[1
]}

机构：

[1] Univ Illinois, Dept Med, Div Hematol & Oncol, Chicago, IL 60612 USA

来源：

JOURNAL OF CLINICAL MEDICINE | 2019年 / 8卷 / 10期

关键词：

sickle cell disease; transplantation; BONE-MARROW-TRANSPLANTATION; QUALITY-OF-LIFE; MATCHED RELATED DONORS; PULMONARY-HYPERTENSION; REDUCED-INTENSITY; UNITED-STATES; RISK-FACTORS; HEALTH-CARE; MORTALITY; ANEMIA;

D O I：

10.3390/jcm8101565

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Sickle cell disease (SCD) is an inherited red blood cell disorder that leads to substantial morbidity and early mortality. Acute and chronic SCD-related complications increase with older age, and therapies are urgently needed to treat adults. Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative therapy, but has been used less frequently in adults compared to children. This is, in part, due to (1) greater chronic organ damage, limiting tolerability to myeloablative conditioning regimens, (2) a higher rate of HSCT-related complications in adults versus children with SCD, and (3) limited coverage by public and private health insurance. Newer approaches using nonmyeloablative and reduced-intensity conditioning HSCT regimens have demonstrated better safety and tolerability, with high rates of stable engraftment in SCD adults. This review will focus on the impacts of HSCT, using more contemporary approaches to SCD-related complications in adults.

引用

页数：17

共 82 条

[1] Relationship between Mixed Donor-Recipient Chimerism and Disease Recurrence after Hematopoietic Cell Transplantation for Sickle Cell Disease [J].

Abraham, Allistair ;

Hsieh, Matthew ;

Eapen, Mary ;

Fitzhugh, Courtney ;

Carreras, Jeanette ;

Keesler, Daniel ;

Guilcher, Gregory ;

Kamani, Naynesh ;

Walters, Mark C. ;

Boelens, Jaap J. ;

Tisdale, John ;

Shenoy, Shalini .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2017, 23 (12) :2178-2183

[2] Prevention of a first stroke by transfusions in children with sickle, cell anemia and abnormal results on transcranial Doppler ultrasonography [J].

Adams, RJ ;

McKie, VC ;

Hsu, L ;

Files, B ;

Vichinsky, E ;

Pegelow, C ;

Abboud, M ;

Gallagher, D ;

Kutlar, A ;

Nichols, FT ;

Bonds, DR ;

Brambilla, D ;

Woods, G ;

Olivieri, N ;

Driscoll, C ;

Miller, S ;

Wang, W ;

Hurlett, A ;

Scher, C ;

Berman, B ;

Carl, E ;

Jones, AM ;

Roach, ES ;

Wright, E ;

Zimmerman, RA ;

Waclawiw, M ;

Pearson, H ;

Powars, D ;

Younkin, D ;

El-Gammal, T ;

Seibert, J ;

Moye, L ;

Espeland, M ;

Murray, R ;

McKinley, R ;

McKinley, S ;

Hagner, S ;

Weiner, S ;

Estow, S ;

Yelle, M ;

Brock, K ;

Carter, E ;

Chiarucci, K ;

Debarr, M ;

Feron, P ;

Harris, S ;

Hoey, L ;

Jacques, K ;

Kuisel, L ;

Lewis, N .

NEW ENGLAND JOURNAL OF MEDICINE, 1998, 339 (01) :5-11

[3]

ALZAHRANI M, 2017, BLOOD S1, V130

[4] Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel [J].

Angelucci, Emanuele ;

Matthes-Martin, Susanne ;

Baronciani, Donatella ;

Bernaudin, Francoise ;

Bonanomi, Sonia ;

Cappellini, Maria Domenica ;

Dalle, Jean-Hugues ;

Di Bartolomeo, Paolo ;

Diaz de Heredia, Cristina ;

Dickerhoff, Roswitha ;

Giardini, Claudio ;

Gluckman, Eliane ;

Hussein, Ayad Achmed ;

Kamani, Naynesh ;

Minkov, Milen ;

Locatelli, Franco ;

Rocha, Vanderson ;

Sedlacek, Petr ;

Smiers, Frans ;

Thuret, Isabelle ;

Yaniv, Isaac ;

Cavazzana, Marina ;

Peters, Christina .

HAEMATOLOGICA, 2014, 99 (05) :811-820

[5] Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease [J].

Ataga, K. I. ;

Kutlar, A. ;

Kanter, J. ;

Liles, D. ;

Cancado, R. ;

Friedrisch, J. ;

Guthrie, T. H. ;

Knight-Madden, J. ;

Alvarez, O. A. ;

Gordeuk, V. R. ;

Gualandro, S. ;

Colella, M. P. ;

Smith, W. R. ;

Rollins, S. A. ;

Stocker, J. W. ;

Rother, R. P. .

NEW ENGLAND JOURNAL OF MEDICINE, 2017, 376 (05) :429-439

[6] Pulmonary hypertension in patients with sickle cell disease: a longitudinal study [J].

Ataga, Kenneth I. ;

Moore, Charity G. ;

Jones, Susan ;

Olajide, Oludamilola ;

Strayhorn, Dell ;

Hinderliter, Alan ;

Orringer, Eugene P. .

BRITISH JOURNAL OF HAEMATOLOGY, 2006, 134 (01) :109-115

[7] The cost of health care for patients with sickle cell disease [J].

Ballas, Samir K. .

AMERICAN JOURNAL OF HEMATOLOGY, 2009, 84 (06) :320-322

[8] 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

[9] Health-Related Quality of Life after Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Disease [J].

Bhatia, Monica ;

Kolva, Elissa ;

Cimini, Laura ;

Jin, Zhezhen ;

Satwani, Prakash ;

Savone, Mirko ;

George, Diane ;

Garvin, James ;

Paz, Mary Llenell ;

Briamonte, Courtney ;

Cruz-Arrieta, Eduvigis ;

Sands, Stephen .

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 2015, 21 (04) :666-672

[10] HLA-haploidentical bone marrow transplantation with posttransplant cyclophosphamide expands the donor pool for patients with sickle cell disease [J].

Bolanos-Meade, Javier ;

Fuchs, Ephraim J. ;

Luznik, Leo ;

Lanzkron, Sophie M. ;

Gamper, Christopher J. ;

Jones, Richard J. ;

Brodsky, Robert A. .

BLOOD, 2012, 120 (22) :4285-4291

← 1 2 3 4 5 6 7 8 9 →