Hematopoietic Stem Cell Transplantation Dr . Ghassemi A Associate professor of pediatric Hematologist Oncologist & stem cell transplantation

Rationale for HSCT in children Allows delivery of supra-lethal doses of chemotherapy (transplant given as rescue) Donor cells may directly attack cancer cells (anti-cancer effect) Correct congenital defects in bone marrow (replaces defective marrow) Corrects congenital immunodeficiencies (replaces immune system that does not work)

Common goal is cure

Role of transplantation 1) Non malignant disease: replace defective marrow 2) Solid tumors: rescue the marrow aplasia 3) Leukemia: cure in three ways: a) High dose chemotherapy b) Replacing malignant marrow c)GVL

When HSCT is utilized First, it can be intended as a curative treatment acting on the minimal residual malignant cells after conventional therapies Secound,it can be used in refractory or relapsed disease Third, it can be used for the immunological effect of Allo HSCT (GVT).

Indication for HSCT 1) Hematology malignancy: AML,ALL,CML,JMML 2) Solid tumors 3) Hematologic disorders, Thalassemia , Aplastic anemia, Fanconi A, sickle cell A,… 4) Inborn error of metabolism : Osteopetrosis, Mucopolysaccharidosis , ,… 5) Immunodeficiency : SCID,HLH, CGD,… 6) Autoimmune disorders MS Evans Syndrome

The prognosis of malignant solid tumors in children has been improved by multidisciplinary therapy involving surgery, radiotherapy and chemotherapy

High-dose chemotherapy (HDC) followed by autologous (Auto) or allogeneic (Allo) hematopoietic stem cell transplantation (HSCT) has contributed significantly to improvements in the prognosis of high-risk chemosensitive tumors. It has been utilized in many kinds of pediatric malignancies, not only hematopoietic tumors but also solid tumors such as neuroblastoma, brain tumors, Ewing’s sarcoma, osteosarcoma, Wilms’ tumor, rhabdomyosarcoma, hepatoblastoma, and retinoblastoma

When HSCT is utilized First, it can be intended as a curative treatment acting on the minimal residual malignant cells after conventional therapies it can be used in refractory or relapsed disease Third, it can be used for the immunological effect of Allo HSCT (GVT).

Allogenic MRD MORD MURD MMD Haploidentical Autologous Cord blood

. Hepatoblastoma HCT followed by Auto or Allo HSCT for patients with high-risk hepatoblastoma (HB) was tried by several groups. The initial report described three patients with stage II or III HB who achieved CR after receiving Auto HSCT, and one patient with a third relapse showed PR (Hara et al., 1998). HSCT for MRD may improve survival (Yoshinari et al., 1998). Subsequent reports of tandem HSCT for three high-risk patients (metastatic or recurrent disease) showed that they failed to remain in remission (Katzenstein et al., 2002)., a report of a patient with recurrent metastatic HB who received non-myeloablative HSCT from a HLA-matched unrelated donor showed a probable GVT effect with GVHD (Inaba et al., 2006).

2 years old girl, First presentation was abdominal distention from 1month ago T: Grip mixture Sonograohy: Hepatic mass BW= 3950gr

WBC= 8700 HB= 10,7, mcv= 63, mch= 17.6 PLT= 85700 ESR= 34 LDH=1145 SGOT=102, SGPT=25, BHCG= Normal a-FP>12100

Percutaneous liver biopsy: Hepatoblastoma (Epithelial type, embryonal and fetal pattern)

8 Course of: VCR +ADR+ Cisplatin 2 Course: Irinotecan 17/2/1393: Complete resection

در بررسی های سریال 6ماه بعد افزایش الفا فتوپروتین (140)داشت سونوگرافی توده 11 میلی متری کبدی داشت مشاوره جراح کبد

RFA Radiofrequency Ablation RFA is a localized, image-guided treatment that uses thermal energy to remove or destroy tumor tissue. The goal of RFA is to destroy the entire malignant tumor and a surgical margin of 1 cm without damaging surrounding tissue.

Efficacy of RFA for Hepatic Tumors The use of RFA as a treatment for hepatic tumors is relatively safe and effective. In patients with metastatic hepatic lesions, RFA produces significant survival benefits in tumors less than 3 cm

J Pediatr Surg. 2012 Mar;47(3):e7-e12. doi: 10. 1016/j. jpedsurg. 2011 J Pediatr Surg. 2012 Mar;47(3):e7-e12. doi: 10.1016/j.jpedsurg.2011.10.075. Radiofrequency ablation in the treatment of liver tumors in children. van Laarhoven S1, van Baren R, Tamminga RY, de Jong KP. Author information Abstract Hepatoblastoma and liver metastasis of Wilms' tumors are rare hepatic tumors in children. Treatment of both tumors consists of a combination of chemotherapy and liver surgery. Radiofrequency ablation (RFA) is frequently used for the treatment of adult liver tumors but is rarely mentioned as a treatment option in pediatric liver tumors. We present a patient with hepatoblastoma and 1 with liver metastasis from a Wilms' tumor. Both patients were treated according to the latest protocols except that surgery included use of RFA. Both are well and recurrence free 8 and 3 years after surgery. Radiofrequency ablation may be a good addition to the existing arsenal of treatment modalities for pediatric liver tumors. Copyright © 2012 Elsevier Inc. All rights reserved.

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