High‐dose chemotherapy with autologous stem cell rescue for treatment of retinoblastoma: Report of five cases

Abstract

RB is a primarily pediatric cancer arising from the retina, initiated by biallelic loss of the RB1 gene. We report five children with bilateral RB (n = 3), extra-ocular disseminated RB, or disseminated relapsed RB, who were treated with tandem high-dose chemotherapy and autologous stem cell rescue. All patients received at least 2.2 × 10(6) /kg CD34(+) (median, 3.9 × 10(6) /kg) cells. The preparative regimen for course 1 was carboplatin, thiotepa, etoposide, and for course 2, CM and melphalan. ANC of at least 0.5 × 10(9) /L occurred at a median of 11 days (range, 10-12) and 15 days (range, 12-16) after the first and second procedure, respectively. Platelet engraftment occurred at a median of 13 days (range, 12-17) and 15 days (range, 14-22) after the first and second procedure, respectively. All of the five patients treated remain alive and disease free at the last follow-up time, ranging between 21 and 44 months after completion of autologous transplant. Additional therapy was required in one patient, in whom enucleation had to be performed because of early disease relapse, refractory to local therapy. Intensification of chemotherapy with repeated high-dose chemotherapy and autologous rescue appears an acceptable choice in selected cases with bilateral or extra-ocular disease, either recurrent or refractory.

Stem-Cell Therapy Following High-Dose Chemotherapy in Advanced Retinoblastoma: A Systematic Review

“…The cases of second primary malignancy were commonly found in retinoblastoma survivors, with bone as the most common site of occurrence. It was estimated that the cumulative incidence of secondary bone sarcoma was approximately 7% at 20 years of age 25,37,47,59 . This was also supported by our data showing a total of 16 of 160 (10%) patients suffered from secondary malignancy, with osteosarcoma as the most common secondary malignancy found in 12 of 16 (75%) patients, followed by Ewing sarcoma in 2 of 16 (12.5%) patients.…”

“…It was estimated that the cumulative incidence of secondary bone sarcoma was approximately 7% at 20 years of age. 25,37,47,59 This was also supported by our data showing a total of 16 of 160 (10%) patients suffered from secondary malignancy, with osteosarcoma as the most common secondary malignancy found in 12 of 16 (75%) patients, followed by Ewing sarcoma in 2 of 16 (12.5%) patients. The other commonly found secondary malignancies include the soft tissue sarcoma, leukemia,…”

“…This strategy has shown to produce reliable antineoplastic effects and good hematopoietic recovery after bone marrow suppression as a consequence of using chemotherapy and radiation 47 . Moreover, the intensification of chemotherapy was also considered to be of interest to replace radiation 25 . However, the risk of HDCT in the development of secondary malignancies remains controversial.…”

“…60 However, some centers do not routinely examine histopathology before HDCT-SCT. 25,56 High-Dose Chemotherapy Regimen…”

Survival and ocular preservation in a long-term cohort of Japanese patients with retinoblastoma

“…Treatments for metastatic or trilateral disease are problematic for survival [23]. The other issue is the intensity of neoadjuvant chemotherapy for newborns and very young infants.…”