ISSN: 1300-7777 E-ISSN: 1308-5263
Use of gemtuzumab ozogamicin in the treatment of pediatric relapsed/ refractory Acute Myeloid Leukemia [Turk J Hematol]
Turk J Hematol. 2008; 25(1): 36-41

Use of gemtuzumab ozogamicin in the treatment of pediatric relapsed/ refractory Acute Myeloid Leukemia

Elif Ünal, Indira Sahdev
Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Schneider Children's Hospital, Albert Einstein College of Medicine, Long Island Jewish Medical Center, New Hyde Park, NY 11040, USA

Gemtuzumab ozogamicin (GO, MylotargTM) is an antibody-targeted chemotherapy agent that has been studied in acute myeloid leukemia (AML) at first relapse in adults. There is limited experience in pediatric patients. We report six patients with refractory/relapsed CD33+AML who were treated with GO on compassionate-use basis. One patient attained remission. One patient is still alive following hematopoietic stem cell transplantation (HSCT), and one patient died in remission. Two patients were refractory and three patients had a response with <5% blasts in the bone marrow. Fever and chills, hypotension and hypoxia were observed as side effects. Three patients developed veno-occlusive disease (VOD) of the liver. Two of these three patients had persistence of VOD at the time of their deaths. One patient treated postSCT had bone marrow response without VOD. GO should be used cautiously in chemotherapy-refractory AML pediatric patients due to the high incidence of VOD.

Keywords: Gemtuzumab ozogamicin, Mylotarg, pediatric acute myeloid leukemia, sinusoidal obstruction syndrome, veno-occlusive disease


Pediatrik relaps/refraktor akut myeloid lösemi tedavisinde gemtuzumab ozogamisin kullanımı

Elif Ünal, Indira Sahdev
Division of Pediatric Hematology/Oncology and Stem Cell Transplantation, Schneider Children's Hospital, Albert Einstein College of Medicine, Long Island Jewish Medical Center, New Hyde Park, NY 11040, USA

Gemtuzumab ozogamisin (GO, MylotargTM) erişkin relaps akut myeloid lösemi (AML) vakalarında kemoterapötik olarak çalışılmış monoklonal bir antikordur. Çocuk hastalarda deneyim sınırlıdır. Biz, tedavisinde GO kullanılan altı pediatrik relaps/refrakter CD33+AML hastasını sunmak istedik. GO tedavisi sonrası bir hastada remisyon sağlandı, üç hastada kemik iliğinde <%5 blast ile yanıt elde edildi, iki hasta ise refrakterdi. İlaç yan etkisi olarak ateş ve titreme, hipotansiyon ve hipoksi gözlendi. Üç hastada karaciğerde veno-oklusif hastalık gelişti. Bunlardan ikisinde VOD ölüme katkıda bulundu. Bir hasta GO tedavisini hematopoetik kök hücre transplantasyonu sonrasında aldı ve VOD gelişmedi. Gemtuzumab ozogamisin pediatrik relaps/refrakter AML hastalarında VOD riski nedeniyle dikkatli kullanılmalıdır.

Anahtar Kelimeler: Gemtuzumab ozogamisin, Mylotarg, pediatrik akut myeloid lösemi, sinüzoidal obstrüksiyon sendromu, veno-oklusif hastalık


Elif Ünal, Indira Sahdev. Use of gemtuzumab ozogamicin in the treatment of pediatric relapsed/ refractory Acute Myeloid Leukemia. Turk J Hematol. 2008; 25(1): 36-41

Corresponding Author: Elif Ünal, Türkiye


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