Abstracts / Pediatric Hematology Oncology Journal 3 (2018) S1eS6
chemotherapy was administered. HSCT was offered to all the patients after
initial therapy and ALL maintenance chemotherapy for 2 years was
continued in those who declined HSCT.
Results: Nineteen children were included in study with a median age at
diagnosis of eight years (1 year to 18 years). The median total leukocyte
count at presentation was 10,200 (4500 to 500,000). B-myeloid in fifteen,
T-myeloid in two and B/T lymphoid in two children were the immuno-
phenotype documented. Eleven patients received AML induction therapy
and 8 patients received ALL induction therapy. Eight out of thirteen chil-
dren who underwent allogeneic hematopoietic stem cell transplantation
from the best available donor and two of the five patients who did not
undergo HSCT are alive. The overall survival in our cohort was 42.1%. The
most common cause of mortality was relapse (66%) followed by regimen
related toxicity (33%).
Conclusion: Children who had received ALL induction followed by AML
induction had a trend toward improved survival in our series with an
overall survival of 42%. Relapse was the most common cause of mortality
and MRD based individualized therapy would help optimize outcomes.
EFFICACY OF RITUXIMAB IN THE TREATMENT OF PEDIATRIC
AUTOIMMUNE HAEMATOLOGICAL DISORDERS AND MALIGNANCIES e
A SINGLE CENTRE EXPERIENCE*
B. Varshini*, Rani S. Sirisha, V. Sandhya, Kiran G. Chaitanya. Department of
Paediatric Haematology & Oncology, Rainbow Children Hospital, Hyderabad
* Corresponding Author.
E-mail address: dr.varshini89@gmail.com (B. Varshini).
Background: Rituximab is a chimeric monoclonal antibody targeting B
cells expressing CD20, which is used for various autoimmune conditions
and malignancies refractory to first line drugs including autoimmune
haemolytic anemia (AIHA), chronic ITP, Posttransplant lymphoprolifer-
ative disease (PTLD), Burkitt’s leukemia/lymphoma, SLE etc. This study
aims to assess role of rituximab in children aged 1 to 16 years with auto-
immune hematological disordes and malignancies that are refractory to
standard modes of treatment.
Methods: A total of 43 children seen over 5.5yrs, between 1 and 16 years
with different autoimmune conditions and malignancies who failed to
respond to initial treatment were treated with Rituximab at standard dose
of 375 mg/m2 weekly. Response was defined by remission of symptoms,
complete or partial reduction in requirement of immunosuppressive
drugs.
Results:

Total 43 children (male-21, female-22) were screened during
study which include Hemolytic anemia (18/43, 41.8%) (AIHA
were 13, EVAN syndrome were 2, Other anemia 2), ITP(14/
43,32.5%), Juvenile SLE (5/43,11.6%) and Lymphoreticular ma-
lignancy (7/43,16%).

In hemolytic anemia group 15/18(83.3%) children had complete
response (CR) at 2 months after treatment and 16/18 (89%)
after 6 months and all except one became transfusion free.
Median haemoglobin raise from 5.6 g/dl to 9.3 g/dl was noted
at 2months followup.

In ITP group, 3/14 (21.4%) had CR and 4/14 (28.5%) had Partial
Response (PR) at 2 month follow up. At 6 months follow up 4/
14 (28.5%) had CR.

Among SLE, CR for proteinuria was observed in 3/5(60%). At 6
months follow up CR was seen in 4/5(80%) with subsequent
minimal usage of immunosuppressants.

Among lymphoreticular malignancies, with rituximab based
chemotherapy CR was observed in 4/7(57%) and no response in
3 children at 2 months and all those 4 children sustained CR.

Except one, immediate complications like allergic reactions
were not seen. Delayed immunosuppression was seen in one
child, requiring IVIG replacement therapy for recurrent
gastroenteritis.
S3
Conclusion: In our observation Rituximab was found to be effective and
safe treatment option for refractory autoimmune hematological disorders
and B-cell lymphoproliferative disorders with minimum side effects.
TWENTY-SEVEN YEAR FOLLOW-UP OF FANCONI ANEMIA FROM REFAIN
(REGISTRY FOR FANCONI ANEMIA IN INDIA)-FINITE DISAPPOINTMENT
AND INFINITE HOPE*
Sheila Mohan*, Revathi Raj, Sarala Rajajee, V. Pushpa, V.B. Rao, Detlev
Schindler, Sat Dev Batish, Arleen Auerbach. Apollo Specialty Hospital,
Chennai, India
* Corresponding author.
E-mail address: vsvmezos97@gmail.com (S. Mohan).
Objective: To study the profile and evolution of Fanconi anemia (FA) and
the impact of hematopoietic stem cell transplantation
Method: Children confirmed to have FA were registered and investigated.
Guidelines were provided to the treating physicians on management and
follow up. The siblings were screened and the families were counseled and
antenatal diagnosis was offered. Hematopoietic stem cell transplantation
was offered to symptomatic FA children.
Results: A total of 253 cases were diagnosed to have FA of which 235
(probands and affected siblings diagnosed by screening) were analyzed
and followed up. There were 216 families. Consanguinity was present in
67% of the families. At presentation 92 % had aplastic bone marrow, 2%
presented with AML and thrombocytopenia each and 4% were hemato-
logically stable. Male:Female ratio was 1.4:1 Average age of presentation
was 7.4yrs. Somatic malformations were present in 98 % and these ranged
from “Walk-In malformations” (easily recognizable to the trained eye
when seen on the first visit) like hyperpigmentation, facial features, thumb
anomalies and stunted growth pattern to obscure and rare defects. All
children with cytopenia were commenced on androgenic steroids and 21%
% remain on androgens with supportive therapy. The mortality has been
high at 60%. Hematopoietic stem cell transplantation was performed from
family and alternate donors in 38 patients and the survival has been 50% .
Prenatal testing was done for 4 families
Conclusion: The clinical spectrum of FA is variable but the predominant
pattern has been the presentation with aplastic anemia with a high mor-
tality. “Walk-In Malformations” were a clue to diagnosis.With the
advancement in supportive care and also with the availability of haplo-
identical stem cell transplantation the future seems hopeful for the pa-
tients with Fanconi anemia.
THE IMPACT OF HYDROXYUREA IN CHILDREN WITH HEMOGLOBIN E
BETA THALASSAEMIA e A LONGITUDINAL STUDY*
Shivani Patel*, V.S. Venkateswaran, S. Meena, R. Nikila, M.R. Kesavan, R.
Nandakumar, Ramya Uppuluri, Revathi Raj. Apollo Cancer Institutes, Tamil
Nadu
* Corresponding author.
E-mail address: shivani2105@yahoo.com (S. Patel).
Background: Hemoglobin E-beta (HbE) thalassemia is the most common
hemolytic anemia in south-east Asia and in India, the highest incidence is
seen in the Northeast region. The clinical and hematologic presentation of
HbE thalassemia varies widely. Pharmacological agents like hydroxyurea
which induce fetal hemoglobin production have been found to reduce
transfusion requirement in these patients. The aim of this study is to assess
the response to early introduction of hydroxyurea in children with HbE
beta thalassaemia.
Methods: A longitudinal follow up study was performed at a tertiary care
center in south India which included children upto the age of 18 years of
age with a diagnosis of hemoglobin E beta thalassemia. Children were
classified using Mahidol scoring system into mild, moderate and severe
phenotype based on their clinical and laboratory assessment at every
follow-up. The treatment was based on severity. All children with mild
disease were started on hydroxyurea at a starting dose of 10mg/kg/day and
increased to 20mg/kg/day as tolerated. Children with moderate phenotype
S4 Abstracts / Pediatric Hematology Oncology Journal 3 (2018) S1eS6
were started on hydroxyurea and given packed red cell transfusion if he-
moglobin is less than 7gm%. Those with the severe disease were recom-
mended regular transfusion and iron chelation. Blood counts were
monitored every four weeks, initially and hydroxyurea was withheld if
total white cell count is less than 4000 per cm. Liver and renal function
tests were performed during their annual visit. Hydroxyurea was
commenced in all children above nine months of age.
Results: A total of 80 children were included in the study from January
2002 to August 2018. The male: female ratio was 1.5:1. The median age at
onset of anemia and clinical symptoms was three years (range e 6 months
to 13 years). The median age at first transfusion was two years (6 months e
13 years). At the first visit, the children were classified as per the Mahidol
scoring system, and the mild phenotype was seen in 51% children, mod-
erate in 33% and severe in 16% of patients.
A total of 57 (72%) children were started on hydroxyurea, and 23 (28%)
were advised regular blood transfusion and iron chelation. At the time of
follow up, 4 out of 23 patients who were advised regular blood transfusion
became transfusion independent. Hydroxyurea was started in 58 patients
and 15 (25%) patients did not require any further transfusion on follow up.
Out of all children who were started on hydroxyurea, 58% followed their
baseline growth centile curve, 29% moved to higher centile and 12%
dropped from baseline. In none of the children, severe cytopenias, infec-
tion or other side effects of hydroxyurea was observed.
Treatment characteristics of patients at first visit:
N¼80 (%) Hydroxyurea Transfusions
Mild 41 (51%) 39 (95%) 2 (5%)
Moderate 26 (33%) 18 (73%) 8 (26%)
Severe 13 (16%) 0 13 (100%)
Treatment characteristics after starting hydroxyurea:
N¼80 (%) Hydroxyurea Transfusions
Mild 48 (60%) 45 (94%) 3 (6%)
Moderate 20 (25%) 9 (45%) 11 (55%)
Severe 12 (15%) 0 12 (100%)
Conclusion: Hydroxyurea is a safe drug to use in children as young as 9
months and should be recommended even in asymptomatic children.
Hydroxyurea helps reduce the need for transfusion in the moderately se-
vere category and does not increase the risk of infection. Hydroxyurea
helps maintain hemoglobin in all mild phenotypes and reduce transfusion
requirement in 25% of patients with moderate to the severe phenotype.
Children with the severe phenotype will benefit from optimal transfusion
and chelation program.
DONOR LYMPHOCYTE INFUSION IN CHILDREN e AN OUNCE OF
PREVENTION PROVIDES A POUND OF CURE*
V.S. Venkateswaran*, S. Meena, P. Shivani, M.R. Kesavan, Nikila, U.
Ramya, V. Lakshman, R. Revathi. Department of PediatricHematology,
Oncology and Blood and Marrow Transplantation, Apollo Cancer Institutes,
Teynampet, Chennai, Tamilnadu, India
* Corresponding author.
E-mail address: vsvmezos97@gmail.com (V.S. Venkateswaran).
Background: Donor lymphocyte infusion (DLI) is a form of cellular
immunotherapyeffective in preventing a relapse due to graft versus
leukaemia effect in malignancies and prevents the progression of
impending graft rejectionin benign hematological conditions. There is
scant data for its use in children and we describe the indications and cost
effective methods of delivering care at our centre.
Patients and methods: We conducted a retrospective analysis of the
children who underwent HSCT and received DLI at Apollo Cancer Institutes
from January 2011 to May 2018. The desired volume of fresh peripheral
blood from the donor was infused based on the CD3 count in a graded
regimen with the cell dose of 1 x 10*5 CD3 cells/kg (1 x 10*4 CD3/kg in
haplo transplants), 5 x 10*5 cD3 cells/kg, 1 x 10*6 CD3 cells/kg depending
on the graft kinetics and the clinical status.
Results: Five sixty ninechildren underwent HSCT and DLI was performed
in 58 children (M:F- 1.4: 1) with72.4% were for benign haematological
conditions. 12% children received reduced intensity conditioning.88% had
fully matched donor and 12% hadhaplo-identical family donor. Peripheral
blood stem cellswas used in 62%and bone marrow in 38% children. DLI to
prevent graft rejection was given in 79% and as pre-emptive therapyin 21%
children withleukemia to prevent a molecular relapse. In our cohort,28
children (48%) received single DLI, two DLI in 17 (29%) and three in 13
children(23%). DLI prevented graft rejection / relapse in 72% of which 21
children(46%) achieved 100% chimerism, 12 children(26%) had mixed
chimerism and were clinically stable. 22out of 58children developed mild
skin and mouth GVHD and gut involvement was seen only in four. The
mortality was 17% (10/58) due to graft loss, relapse of leukemia and one
attributed to GVHD.
Conclusion: DLIis an effective tool andsafe procedure andrequired very
small amounts of peripheral blood from 0.1 ml to 75 ml. Careful follow up
of graft kinetics and clinical vigilance for grade 4 GVHD makes it an easily
applicable tool even in resource constrained settings and in haploidentical
HSCT.
REPORTING FIRST SERIES OF TCR АbAND CD45 RA DEPLETED
PAEDIATRIC HEMATOPOEITIC STEM CELL TRANSPLANTS IN INDIA*
Ravi Joshi*, Pooja Mallya, Shobha Badiger, P. Vasundhara, M.V.
Archana, Mohammad Salman, Mohan Reddy, Sunil Bhat. Mazumdar
Shaw Cancer Center, Narayana Health City, Pediatric Hematology Oncology,
Bangalore, India
* Corresponding author.
E-mail address: Drravijoshijkd@gmail.com (R. Joshi).
Introduction: T-cell depletion either ex vivo/in vivo effectively decreases
the incidence of severe graft-versus-host disease in recipients of HLA-
mismatched allografts. However, T cell depletion is associated with
delayed immune recovery and increased infections.CD45RA depletion
provides a large number of donor memory T cells to the recipients and is
associated with enhanced early T-cell recovery and provides effective
protection against viremia.
Materials And Methods: Retrospective data was collected from hospital
records onCD45 RA depletion transplants from May 2018 till Aug
2018.Data regarding diagnosis, conditioning regimen, cell dose used, time
of neutrophil and platelet engraftment, incidence of GVHD, viremia and
outcome was collected and analyzed.
Results: Total 6 cases of TCR ab and CD45 RA cell depletion transplants
were done in the study period. Demographic data, cell doses and various
outcome measures are mentioned in table 1. Profound depletion of
CD45RA+ and TCR ab T cells was reliably obtained in all products on
ClinimacsMilteny device.All patients engrafted except one who is still
day+10 .Neutrophil engraftment ranged from day+9 to day +13, platelet
engraftment ranged from day+8 to day+14.None of the patients had acute
GVHD.Of 5 patients who engrafted ,4 had excellent chimerism at their last
follow up.1 patient of Fanconi anemia rejected at day+80, now undergoing
second transplant. Only 1 patient had cytomegalovirus viremia with 10457
copies per ml and had bilateral pneumonia requiring PICU admission. She
was started on InjGanciclovir.Now she is better and recovered from illness.
Immune reconstitution monitored by immunophenotyping(IPT) after 3
weeks.3 patients had natural killer cell(NK) recovery and it needs further
monitoring.
Conclusion:The technique was feasible and very well tolerated in our
patients with no acute GVHD till last follow up (day+93).The incidence of
viremia reduced significantly. This technique provides large memory-T-
cell doses, which may facilitate engraftment. Collectively, this novel
approach may facilitate rapid immune reconstitution and prevent viral
infections. Additional trials in thalassemia and other disease groups will be