EBMT 2014 - Physicians Abstract Submission

Disease-specific topics
Haemoglobinopathy and Inborn errors of metabolism
EBMT2014-PHYSCANS-1959
HAEMATOPOIETIC STEM CELL TRANSPLANTATION FOR SICKLE CELL DISEASE: AN INTERNATIONAL
SURVEY ON BEHALF OF EUROCORD-MONACORD, EBMT PAEDIATRIC DISEASE WORKING PARTY AND
CIBMTR
Eliane Gluckman
1,*
, Annalisa Ruggeri
1
, Myriam Labopin
2
, Fernanda Volt
1
, Agnes Devergie
1
, Marina Cavazzana
3
,
Barbara Cappelli
4
, Mary Eapen
5
, Belinda Simoes
6
, Susanne Matthes
7
, Vijay Ramanan
8
, Jean Hugues Dalle
9
, rene
Roberts
10
, Josu de la Fuente
10
, Vanderson Rocha
11
, Franco Locatelli
12
, Christina Peters
13
, Francoise Bernaudin
14
1
Eurocord-Monacord,
2
ALWP EBMT,
3
BMT Necker H, Paris, France,
4
Eurocord-Monacord, Monaco, Monaco,
5
CBMTR,
Milwaukee, United States,
6
USP, Riberao Preto, Brazil,
7
St Anne, Vienna, Austria,
8
BMT unit, Pune, ndia,
9
Robert Debre
, Paris, France,
10
mperial College, London, United Kingdom,
11
Eurocord APHP, Paris, France,
12
Ospedale Bambino Gesu
, Roma, taly,
13
St Anna Children Hospital, Vienna, Austria,
14
CHC , Creteil, France
Preferred method of presentation: Oral Only
Introduction: Sickle cell disease (SCD) is the most common inherited haemoglobinopathy, and it is associated with numerous
complications and early mortality. So Iar, the only curative therapy Ior SCD is allogeneic hematopoietic stem cell transplantation
(HSCT). However, only a minority oI eligible SCD patients (pts) underwent the procedure, especially when a matched sibling donor
(MSD) was not available. A consensus on indications Ior HSCT was recently reached; however, there are still concerns on unrelated
HSCT because oI the associated risks. Another relevant barrier Ior HSCT in SCD patients is the lack oI suitable matched unrelated
donors (MUD), due to the ethnic origin oI most patients. Conversely, data on the use oI alternative donors such as cord blood (CB) or
related haploidentical donors are scarce.
MateriaIs (or patients) and methods: The aim oI this survey is to describe HSCT Ior SCD initially in Europe, and later in other
areas with the Iinal goal oI increasing the awareness and extending the use oI this procedure to a higher number oI SCD pts. A
preliminary survey Irom Eurocord-EBMT and CIBMTR identiIied 1235 pts transplanted Ior SCD with an overall survival (OS) oI
96. 608 were reported Irom the EBMT (BM Irom MSD, n487, CB Irom MSD, n 70, MUD, n17, haploidentical donors, n34)
and 627 Irom the CIBMTR (BM Irom MSD, n430, CB Irom MSD, n 71, MUD, n61, haploidentical donors, n65). The results
below are Irom the retrospective analysis oI the European cohort.
ResuIts: We analyzed data Irom 608 pts (93 children 18 years) transplanted (1
st
allogeneic graIt) Ior SCD in 92 EBMT centres
Irom 1986 to 2013, and reported to EBMT/Eurocord. The majority oI the HSCT were perIormed in France (38), Iollowed by
Belgium, Italy and UK. Median age at transplant was 8.9 years (0.10-48). Median Iollow-up was 28 months (3-273). Myeloablative
conditioning (CT) regimen was used in 94 oI cases, and the most Irequent CT used was busulIan(BU)cyclophosphamide(CY)
(76), Iollowed by BUIludarabine(FLU) (15), FLUtreosulIan (6) and FLUmelphalan (3). TBI was included in 2 oI the
CT and ATG in 67. Most pts received cyclosporine (CsA) associated with methotrexate (43) or CsA alone (20) Ior GVHD
prophylaxis.
The cumulative incidence (CI) oI neutrophil engraItment at day60 was 97 (98 Ior MSD, 94 Ior related CB, 100 Ior MUD
and 72 Ior haplo transplants) with a median time oI 19 days. Three percent (n15) oI pts Iailed to engraIt and 2 (n9) had
secondary graIt Iailure. Grade II-IV acute GVHD occurred in 16 oI pts and chronic GVHD in12.
Two-year OS was 94 (94 Ior MSD, 98 Ior related CB, 91 Ior MUD and 78 Ior haplo transplants). Thirty-six pts died, and
the reported causes oI death were GVHD (28), inIection (28), sinusoidal obstruction syndrome (6), hemorrhage (8) and other
causes (30).
Discussion: The Iindings oI this survey provide an overview oI HSCT outcomes Ior SCD and may serve to assist medical
proIessionals to improve criteria and indications oI HSCT Ior SCD patients. This excellent results might be Iurther improved iI
patients undergo allogeneic HSCT with less comorbities and with contemporary conditioning regimen and suIIicient GVHD- and
rejection prophylaxis. Also, it might serve as basis Ior the expansion oI HSCT Ior SCD worldwide and to provide data Ior new
prospective protocols.
DiscIosure of Interest: None Declared
Keywords: allogeneic hematopoietic stem cell transplantation, sickle cell disease