Transplantation of Hematopoietic Stem Cells for Primary Immunodeficiencies in Brazil: Challenges in Treating Rare Diseases in Developing Countries
Carregando...
Citações na Scopus
13
Tipo de produção
article
Data de publicação
2018
Título da Revista
ISSN da Revista
Título do Volume
Editora
SPRINGER/PLENUM PUBLISHERS
Autores
NICHELE, Samantha
DAUDT, Liane E.
TAVARES, Rita B.
SEBER, Adriana
KERBAUY, Fabio R.
KOLISKI, Adriana
LOTH, Gisele
VIEIRA, Ana K.
DARRIGO-JUNIOR, Luiz G.
Citação
JOURNAL OF CLINICAL IMMUNOLOGY, v.38, n.8, p.917-926, 2018
Resumo
The results of hematopoietic stem cell transplant (HSCT) for primary immunodeficiency diseases (PID) have been improving over time. Unfortunately, developing countries do not experience the same results. This first report of Brazilian experience of HSCT for PID describes the development and results in the field. We included data from transplants in 221 patients, performed at 11 centers which participated in the Brazilian collaborative group, from July 1990 to December 2015. The majority of transplants were concentrated in one center (n=123). The median age at HSCT was 22months, and the most common diseases were severe combined immunodeficiency (SCID) (n=67) and Wiskott-Aldrich syndrome (WAS) (n=67). Only 15 patients received unconditioned transplants. Cumulative incidence of GVHD grades II to IV was 23%, and GVHD grades III to IV was 10%. The 5-year overall survival was 71.6%. WAS patients had better survival compared to other diseases. Most deaths (n=53) occurred in the first year after transplantation mainly due to infection (55%) and GVHD (13%). Although transplant for PID patients in Brazil has evolved since its beginning, we still face some challenges like delayed diagnosis and referral, severe infections before transplant, a limited number of transplant centers with expertise, and resources for more advanced techniques. Measures like newborn screening for SCID may hasten the diagnosis and ameliorate patients' conditions at the moment of transplant.
Palavras-chave
Primary immunodeficiencies, stem cell transplantation, severe combined immunodeficiency, Wiskott-Aldrich syndrome, Latin America
Referências
- Antoine C, 2003, LANCET, V361, P553, DOI 10.1016/S0140-6736(03)12513-5
- BACH FH, 1968, LANCET, V2, P1364
- Baker MW, 2009, J ALLERGY CLIN IMMUN, V124, P522, DOI 10.1016/j.jaci.2009.04.007
- Bolanos-Meade J, 2012, BLOOD, V120, P4285, DOI 10.1182/blood-2012-07-438408
- Bonfim C, 2017, BIOL BLOOD MARROW TR, V23, P310, DOI 10.1016/j.bbmt.2016.11.006
- Brodsky RA, 2008, BONE MARROW TRANSPL, V42, P523, DOI 10.1038/bmt.2008.203
- Buckley RH, 1999, NEW ENGL J MED, V340, P508, DOI 10.1056/NEJM199902183400703
- Carneiro-Sampaio M, 2013, J CLIN IMMUNOL, V33, P716, DOI 10.1007/s10875-013-9865-6
- Condino-Neto A, 2015, ALLERGOL IMMUNOPATH, V43, P493, DOI 10.1016/j.aller.2014.05.007
- Condino-Neto A, 2014, CLIN EXP IMMUNOL, V178, P16, DOI 10.1111/cei.12495
- Costa-Carvalho B, 2017, EXPERT REV CLIN IMMU, V13, P483, DOI 10.1080/1744666X.2017.1255143
- de la Morena MT, 2017, J ALLERGY CLIN IMMUN, V139, P1282, DOI 10.1016/j.jaci.2016.07.039
- de Oliveira EB, 2015, PEDIATR BLOOD CANCER, V62, P2101, DOI 10.1002/pbc.25674
- Dvorak CC, 2013, J CLIN IMMUNOL, V33, P1156, DOI 10.1007/s10875-013-9917-y
- Eapen M, 2012, BIOL BLOOD MARROW TR, V18, P1438, DOI 10.1016/j.bbmt.2012.03.003
- Espinosa-Rosales FJ, 2016, J CLIN IMMUNOL, V36, P415, DOI 10.1007/s10875-016-0277-2
- Esteves I, 2015, BONE MARROW TRANSPL, V50, P685, DOI 10.1038/bmt.2015.20
- Fasth A, 2009, AM J HEMATOL, V84, P469, DOI 10.1002/ajh.21454
- Fernandes JF, 2012, BLOOD, V119, P2949, DOI 10.1182/blood-2011-06-363572
- Gale RP, 2016, BONE MARROW TRANSPL, V51, P898, DOI 10.1038/bmt.2016.35
- Gaspar HB, 2013, BLOOD, V122, P3749, DOI 10.1182/blood-2013-02-380105
- GATTI RA, 1968, LANCET, V2, P1366
- Gennery AR, 2010, J ALLERGY CLIN IMMUN, V126, P602, DOI 10.1016/j.jaci.2010.06.015
- GLUCKSBERG H, 1974, TRANSPLANTATION, V18, P295, DOI 10.1097/00007890-197410000-00001
- Griffith LM, 2016, J ALLERGY CLIN IMMUN, V138, P375, DOI 10.1016/j.jaci.2016.01.051
- Griffith LM, 2014, J ALLERGY CLIN IMMUN, V133, P335, DOI 10.1016/j.jaci.2013.07.052
- Gungor T, 2014, LANCET, V383, P436, DOI [10.1016/s0140-6736(13)62069-3, 10.1016/S0140-6736(13)62069-3]
- Hassan A, 2012, BLOOD, V120, P3615, DOI 10.1182/blood-2011-12-396879
- Jaimovich G, 2017, BONE MARROW TRANSPL, V52, P798, DOI 10.1038/bmt.2017.48
- Kanegae Marilia Pyles P., 2017, Rev. paul. pediatr., V35, P25, DOI 10.1590/1984-0462/;2017;35;1;00013
- Klein OR, 2016, BIOL BLOOD MARROW TR, V22, P895, DOI 10.1016/j.bbmt.2016.02.001
- Kwan A, 2013, J ALLERGY CLIN IMMUN, V132, P140, DOI 10.1016/j.jaci.2013.04.024
- Leiva LE, 2007, J CLIN IMMUNOL, V27, P101, DOI 10.1007/s10875-006-9052-0
- Luznik L, 2001, BLOOD, V98, P3456, DOI 10.1182/blood.V98.12.3456
- Luznik L, 2008, BIOL BLOOD MARROW TR, V14, P641, DOI 10.1016/j.bbmt.2008.03.005
- Marciano BE, 2014, J ALLERGY CLIN IMMUN, V133, P1134, DOI 10.1016/j.jaci.2014.02.028
- Mazzucchelli JTL, 2014, J INVEST ALLERG CLIN, V24, P184
- Moratto D, 2011, BLOOD, V118, P1675, DOI 10.1182/blood-2010-11-319376
- Morillo-Gutierrez B, 2016, BLOOD, V128, P440, DOI 10.1182/blood-2016-03-704015
- Ouederni M, 2016, J CLIN IMMUNOL, V36, P437, DOI 10.1007/s10875-016-0293-2
- Ozsahin H, 2008, BLOOD, V111, P439, DOI 10.1182/blood-2007-03-076679
- Pai SY, 2014, CURR OPIN ALLERGY CL, V14, P521, DOI 10.1097/ACI.0000000000000115
- Pai SY, 2014, NEW ENGL J MED, V371, P434, DOI 10.1056/NEJMoa1401177
- Parta M, 2015, J CLIN IMMUNOL, V35, P675, DOI 10.1007/s10875-015-0204-y
- Kanegae MPP, 2016, J PEDIAT-BRAZIL, V92, P374, DOI 10.1016/j.jped.2015.10.006
- Picard C, 2018, J CLIN IMMUNOL, V38, P96, DOI 10.1007/s10875-017-0464-9
- Schuetz C, 2014, BLOOD, V123, P281, DOI 10.1182/blood-2013-01-476432
- Shah NN, 2017, BIOL BLOOD MARROW TR, V23, P980, DOI 10.1016/j.bbmt.2017.03.016
- Shekhovtsova Z, 2017, HAEMATOLOGICA, V102, P1112, DOI 10.3324/haematol.2016.158808
- Shin CR, 2012, BONE MARROW TRANSPL, V47, P1428, DOI 10.1038/bmt.2012.31
- Slack J, 2017, J ALLERGY CLIN IMMUN, VS0091-6749, P30567
- STORB R, 1983, ANN INTERN MED, V98, P461, DOI 10.7326/0003-4819-98-4-461