Bleesing, J.J.

Davies, S.M.

Filipovich, A.H.

Grimley, M.S.

Jodele, S.

Jordan, M.B.

Kumar, A.

Lawrence, J.M.

Marsh, R.A.

Mehta, P.A.

Myers, K.C.

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High Dose Methylprednisolone for Veno-Occlusive Disease of the Liver in Pediatric Bone Marrow Transplant Recipients 

S312 Poster Session Iactivities. More than 2/3 of PHO providers stated that they pro-
vide BMT services at their institutions, most commonly inpatient
coverage (73%). The proportion of providers exposed to BMT
early in their training was significantly higher amongst BMT pro-
viders than PHO providers (51% vs. 18% during medical school
[p\0.0001] and 70% vs. 50% during residency [p\ 0.005]). Ex-
posure during fellowship (94%) did not differ amongst groups.
The decision to pursue a career in BMT was made before fellow-
ship (medical school or residency) in 50% of the respondents. A
lower proportion of BMT providers reported currently being in-
volved in education of medical students and residents compared
to PHO providers (98% vs. 76%, p\0.0001). Of 53 trainees,
64% reported that they were not contemplating a career in
BMT. Of these, 68% stated that inadequate exposure to BMT
prior to PHO fellowship was the reason. Only 26% reported
BMT exposure in medical school and 43% during residency.
The two most common reasons for the choice of a BMT career
were the degree of intellectual and scientific challenge (89%)
and role models/mentors in the field (67%). This survey suggests
that early exposure to BMT during medical school and residency
results in increased interest in pursuing a career in BMT. BMT
physicians and training program directors can foster interest in
the BMT field by promoting BMT focused education and clinical
inpatient and outpatient rotations during medical school and res-
idency. This early exposure to BMT may aid in a higher recruit-
ment of future transplant providers.289
HIGH DOSE METHYLPREDNISOLONE FOR VENO-OCCLUSIVE DISEASE OF
THE LIVER IN PEDIATRIC BONE MARROW TRANSPLANT RECIPIENTS
Myers, K.C.1, Bleesing, J.J.1, Filipovich, A.H.1, Marsh, R.A.1,
Jordan, M.B.1, Kumar, A.1, Grimley, M.S.1, Lawrence, J.M.2,
Mehta, P.A.1, Davies, S.M.1, Jodele, S.1 1Cincinnati Children’s Hospital
Medical Center, Cincinnati, OH; 2Cincinnati Children’s HospitalMedical
Center, Cincinnati, OH
Veno-occlusive disease (VOD) of the liver is a well-recognized se-
rious complication of stem cell transplantation (SCT) with very few
successful treatment modalities available for severe disease. While
there have been some reports demonstrating success in adults with
high dose steroid therapy, experience in the pediatric population is
lacking. In 587 consecutive SCT patients treated at our institution
since 2003 we retrospectively identified 14 (2.4%) who developed
VOD.Of these 8 (57%) were treated with high-doseMethylprednis-
olone (500 mg/m2/dose every 12 hours for 6 doses). Steroid therapy
was initiated at or prior to first ultrasound evidence of reversal of
portal venous flow, and prior to meeting criteria for initiation of de-
fibrotide therapy. Four of these patients were also treated with De-
fibrotide starting 2 to 5 days following initiation of steroids. Seven
patients with VOD were diagnosed with multi-organ failure
(MOF) due to renal impairment and pulmonary dysfunction attrib-
utable to fluid overload or mechanical impingement from abdominal
distention or hepatic enlargement. Four of these 7 patients required
renal replacement therapy (RRT) and 5 required ventilator support
(see Table). Response to high dose steroid therapy as defined by de-
crease in bilirubin by 50% by ten days from therapy initiation wasTable.
Patient / Dgn Donor (match) Age (years) Portal System Doppler Days
1 / PPB Auto 7 Reversal of Flow
2 / CGD URD (7/8) 10.6 Reversal of Flow
3 / RB Auto 0.7 Reversal of Flow
4 / ALL MSD (8/8) 16.2 Reversal of Flow
5 / FA MUD (8/8) 6.3 Reversal of Flow
6 / NB Auto 6.6 Reversal of Flow
7 / ALL MUD (8/8) 4.8 Reversal of Flow
8 / DKC MSD (8/8) 1.2 Reversal of Flow
BMT indicates bone marrow transplant; DKC, dyskeratosis congenita; MSD, m
tologous stem cell transplant; ALL, acute lymphoblastic leukemia; MUD, matche
ulomatous disease; URD, unrelated donor; **RRTand ventilator support, *vennoted in 5/8 (63%) patients, occurring within 3 and 6 days of steroid
therapy. Two patients died due to VODwithMOF. Six survivors re-
covered from VOD at the median 15d (range 4-40) from VOD diag-
nosis. Overall survival as a groupwas 75%; however survival amongst
responders was 100%. No serious toxicities related to high dose ste-
roid therapy, such as refractory hypertension, increased infection, or
behavioral disturbances were observed. Transient hyperglycemia
was seen in some patients. We conclude that high dose steroid ther-
apy if initiated early may reverse VOD of the liver in pediatric SCT
patients, abrogating the need for defibrotide therapy with its associ-
ated toxicities and regulatory difficulties. Further prospective clinical
trials are required to assess the true efficacy of high dose steroid ther-
apy for post SCT VOD.290
BONE HEALTH IN CHILDREN AND ADOLESCENTS UNDERGOING HEMA-
TOPOIETIC CELL TRANSPLANTATION IN A SINGAPORE CENTER
Goh, Z.3, Low, S.L.2, Khanlian, C.1, Mohamed Ismail, N.D.B.2,
Chua, H.C.1, Villegas, M.S.1, Lee, Y.S.1, Tan, P.L.1 1National Univer-
sity Health System, Singapore; 2National University Health System, Sin-
gapore; 3Yong Loo Lin School of Medicine, National University of
Singapore, Singapore
We retrospectively reviewed 103 pediatric patients transplanted
between 2001-2010 to evaluate the prevalence of low bone mineral
density (BMD). Areal BMD of the lumbar spine and whole body
were determined by dual energy X-ray absorptiometry (DXA) im-
aging. Z-scores calculated were standardized by age and gender
based on a reference population. BMD was defined as normal if
Z-score . -1.0; borderline low if -2.0 \ Z-score # -1.0; low if
Z-score # -2.0; and osteoporosis if Z-score # -2.0 with a history
of fracture. Spine Z-scores were used in analysis. Only 45 of the
103 patients had any BMD records. Of these, 18 had serial
BMD evaluations. The median age at time of hematopoietic cell
transplant (HCT) was 10.2 (range, 0.1-16.8) years. Forty-three pa-
tients had allogeneic (4 had 2 HCT) while 2 had autologous HCT.
Majority (N 5 30) received non-radiation based preparative regi-
men; the commonest indication being a hematologic malignancy
(N 5 33). Majority (N 5 33) had graft versus host disease; 27 of
whom required systemic steroids. Of the patients who had pre
HCT BMD, 45% (5 of 11) already had abnormal Z-scores. Trend-
ing of BMD was possible in 17 of the 45 patients who had more
than one BMD. Recovery from a nadir Z-score was noted in
40% (7 of 17) of these patients at the time of analysis. Of the pa-
tients who had post HCT BMD, 23% (9 of 39) and 28% (11 of 39)
had borderline low and low Z-scores, respectively. One of these 20
patients (5%) had clinically significant fractures fulfilling the
WHO criteria for osteoporosis. Musculoskeletal pains were re-
ported by 29% (13 of 45) of patients but an abnormal Z-score
was noted in only 60% (8 of 13). Endocrinopathies including sex
hormones and thyroid function deficiencies was noted in 13% (6
of 45) of patients. The 3 patients with hypogonadism also had ab-
normal BMD and required hormonal replacement. One of these 3
patients received an oral bisphosphonates. Majority had calcium/
vitamin D supplements and about half (19 of 45) had physiother-
apy to optimize bone health. Despite the limitations known offrom BMT to VOD Therapy Multiorgan Failure Status
+31 Steroids Yes Alive
+11 Steroids No Alive
+14 Steroids Yes* Alive
+16 Steroids/Defibrotide Yes** Alive
+15 Steroids/Defibrotide Yes** Dead
+13 Steroids/Defibrotide Yes** Alive
+12 Steroids Yes Alive
+5 Steroids/Defibrotide Yes** Dead
atched sibling donor; NB, neuroblastoma; RB, retinoblastoma; Auto, au-
d unrelated donor; PPB, pleuropulmonary blastoma; CGD, chronic gran-
tilator support.


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High Dose Methylprednisolone for Veno-Occlusive Disease of the Liver in Pediatric Bone Marrow Transplant Recipients

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