Pediatric Hematopoietic Stem Cell Transplant - Experience of an Indian Tertiary Care Center

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Pediatric Hematopoietic Stem Cell Transplant - Experience of an Indian Tertiary Care Center Dr Chirag A Shah Diplomate American Board of Hematology and Medical Oncology Director, Dept of Hemato-Oncology and Stem Cell Transplant APOLLO CBCC Cancer Care, Ahmedabad, Gujarat

Introduction Considerable progress has been made in HSCT for pediatric malignant and nonmalignant conditions in terms of: Type of pediatric diseases successfully treated with HSCT Use of alternative donor stem-cell sources New conditioning regimens Awareness of late effects in pediatric HSCT recipients

We present our transplant data pertaining to the pediatric HSCT done at our center from Jan 2008 till date

Methods Patients aged less than 18 years of age at the time of HSCT were defined as pediatric population and included in the analysis All the patients irrespective of diagnosis were included in the analysis

Conditioning regimen Variety of conditioning regimen were used such as: depending on disease and patient specific issues 1. BuCy (Busulfan, cyclophosphamide) 2. Flu-Mel (fludarabne, melphalan) 3. Bu-Flu (busulphan, fludarabine) 4. TTF (treosulphan, thiotepa and fludarabine) 5. CyATG (cyclophosphamide, ATG)

Facility All patients underwent transplant in High efficiency particle air filtration (HEPA) unit equipped with positive pressure ventilation (PPV) Transplant unit and nursing team are dedicated - not used for other purpose Many of the nursing staff and doctors are working in unit for over 5-7 years

Stem Cell Source All of the patients received filgrastim (GSCF) mobilized peripheral blood stem cell (PBSC) graft, except one, for whom bone marrow stem cells were collected

Results Data of patients with both, malignant and non-malignant hematological illnesses was analyzed in terms of 1. rates of Engraftment 2. Day 100 survival 3. Disease free survival.

Allogeneic & Autologus Transplant - 24 20 18 18 16 14 12 10 8 6 6 4 2 0 Allogeneic Autologus

Male & Female Transplant Number - 24 20 19 18 16 14 12 10 8 6 5 4 2 0 Male Female

Disease wise Distribution of patients 10 9 9 8 7 7 6 5 4 3 3 2 2 1 1 1 1 0 AML Thalassemia H L Neuroblastoma ALL N H L A A

Haploidentical & Matched Sibling Transplants 12 10 10 8 8 6 4 2 0 Haploidentical transplants (current survival 5 out of 8) Matched Sibling transplants (current survival 8 out of 10)

Survival Data Disease Engraftment Day 100 Survival Long Term Survival AML 67% 83% 56% Thalassemia 100% 100% 86% ALL 100% 100% 100% H L 100% 100% 100% N H L 100% 100% 100% A A 100% 100% 100% Neuroblastoma 100% 100% 50%

Conclusions HSCT in pediatric population is an important treatment modality especially for malignancies such as Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Neuroblastoma. It is also an important treatment for Serious Non malignant diseases like Thalssemia Major, Sickle cell Anemia, Aplastic anemia and others. Children tolerate transplant better than adults, and have significantly better results, including lower mortality, low graft versus host disease.

For non caucasians, finding a match in unrelated donor registries is very uncommon. Haplo-identical transplant is an attractive strategy for patients lacking fully matched donor, especially for high risk patients, in urgent need for transplant, with results comparable to matched unrelated donor transplant.

Results from our center are comparable to those from other centers where pediatric transplants are performed regularly. For diseases such as thalassemia, transplants should be considered early in the course of disease given the better results. Same is applicable to Sickle Cell Disease.

Thank you Dr Chirag A Shah Dr Arun Karanwal Dr Vinay Bohara Dr Ravish Shah Dr Pushkar Srivastava Pediatric Intensivist Ms Upasana, Ms Dhanya, Ms Sonal and Nursing Team; Ms Khyati Many other members of Apollo CBCC Cancer Care Team