Stem cell transplantation for haemophagocytic lymphohistiocytosis in children: experience at two Israeli centres

Ref ID: 18567

Author:

J. Stein (1), A. Kraus (1), J. Yacobovich (1), H. Tamary (1),
Y. Kodman (1), O. Dgani (1), I. Yaniv (1), M. Weintraub (2),
P. Stepensky (2)

Author address:

(1)Schneider Children’s Medical Center, Sackler Faculty of
Medicine, Tel Aviv University (Petach Tikva, IL); (2)Hadassah
University Medical Center (Jerusalem, IL)

Full conference title:

Annual Meeting of the EBMT, 38th

Abstract:

Hemophagocytic Lymphohistiocytosis (HLH) is an aggressive
disorder of immune regulation that carries a grave prognosis;
stem cell transplant (SCT) after induction therapy is the recommended defi nitive treatment. Unrelated donors (UD) are used
for patients (pts) lacking a matched family donor, and for those
lacking a molecular diagnosis (as sibling donors might themselves be genetically at risk but not yet affected). Two pediatric centers collectively transplanted 22 pts with HLH between 1991 and
2011. Etiologies included mutations of Perforin (7 pts), Munc 13
(2), Munc 18 (3), Syntaxin (1), as well as T-cell lymphoma (1) and
EBV infection (1). No molecular or infectious etiology was identi-
fi ed in two extensively evaluated pts, and treatment predated the
availability of molecular diagnosis in 5 pts. Twelve pts were in
complete response at the time of transplant and 6 had less than
a complete response; response data were unavailable in 4 pts.
Unrelated donors were used for 10 children. Grafts were derived
from marrow (12), peripheral blood (8), cord blood (1), or cord
blood + marrow (1). Six pts, mostly transplanted in the 1990’s,
received fully ablative conditioning; others received fl udarabine
containing regimens; 91% received serotherapy during conditioning. Cyclosporine was administered after transplant usually in
combination with either methotrexate or mycophenolate mofetil.
Fifteen patients (68%) are alive (8/10 UD, 7/12 related donors)
without evidence of disease, four of whom have stable mixed
donor chimerism of less than 70%. Four of 6 pts with signs of
active HLH at the time of SCT survive, as did 4/5 children with
HLH involvement of the central nervous system at diagnosis (two
without apparent developmental delay). Fatal cases of pneumonia were caused by CMV (1), RSV (2), and adenovirus together
with aspergillus (1), suggesting that aggressive anti-viral and
anti-fungal prophylaxis is imperative in this high-risk population.
The child with lymphoma died of multi-organ failure 1 day after
transplant, and an additional pt succumbed to pulmonary venoocclusive disease 5 months after SCT. Severe graft vs. host
disease occurred in only one child. SCT from related and unrelated donors using fl udarabine-based reduced intensity conditioning with the addition of serotherapy is an effective treatment
for HLH.

Abstract Number: P1041

Conference Year: 2012

Link to conference website: NULL

New link: NULL

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