High-dose chemotherapy followed by stem cell transplantation in the management of retinoblastoma: a systematic review

Jaradat, Imad; Mubiden, Rasmi; Salem, Ahmed; Abdel-Rahman, Fawzi; Al-Ahmad, Iyad; Almousa, Abdelatif

doi:10.5144/1658-3876.2012.107

Cited by 17 publications

(19 citation statements)

References 21 publications

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“…In patients with tumor at the surgical margin of the optic nerve and/or extra scleral extension, six of seven patients (85.7%) were alive with no evidence of disease at the time of follow‐up. The authors conclude that durable tumor control is possible in patients with non‐CNS metastases and trilateral or bilateral advanced RB, and in patients with tumor at the surgical margin of the optic nerve and/or extra scleral extension using HDCT/AuSCT, and suggest that patients with CNS metastases require thiotepa to improve tumor control .…”

Section: Discussionmentioning

confidence: 94%

“…ANC of at least 0.5 9 109/L occurred at a median of 11 days (range, 10-12) and 15 days (range, 12-16) after the first and second HDCT-AuSCT, respectively. Platelet engraftment, defined as the first of seven consecutive days of a platelet count of at least 20 9 10 9 /L sustained without a transfusion, occurred at a median of 13 days (range: 12-17) and 15 days (range, [14][15][16][17][18][19][20][21][22] after the first and second HDCT-AuSCT, respectively.…”

Section: Resultsmentioning

confidence: 99%

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

Caselli

Tamburini

Torre

et al. 2014

Pediatric Transplantation

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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.

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Section: Discussionmentioning

confidence: 94%

Section: Resultsmentioning

confidence: 99%

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

Caselli

Tamburini

Torre

et al. 2014

Pediatric Transplantation

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“…[2][3][4][5][6][7] However, an appropriate single protocol of HDC has never been assessed. RB is known to respond well to alkylating agents, and Mel is one of the key drugs in this regard.…”

Section: Discussionmentioning

confidence: 99%

High-dose chemotherapy for high-risk retinoblastoma: clinical course and outcome of 14 cases in the National Cancer Center, Japan

Yasui¹,

Kawamoto²,

Fujiwara

et al. 2014

Bone Marrow Transplant

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The prognosis of high-risk retinoblastoma (RB) with extraocular disease, relapse, or invasion of the cut end of the optic nerve is extremely poor. Following the discontinuation of thiotepa production in Japan, BU-and melphalan (Mel)-based regimens have been used, followed by the standard treatment for neuroblastoma. This study retrospectively analyzed 14 high-risk RB patients who underwent high-dose chemotherapy (HDC) and hematopoietic SCT; 8 received a BU/Mel conditioning regimen and 6 received other regimens. The disease status at HDC was relapse in 8 patients and extraocular involvement in 5. All patients received peripheral blood stem cell infusion 41.5 × 10 6 /kg. Engraftment occurred within a median of 11 days (BU/Mel: 10-13, others: 9-13). Primary toxicities included mucositis (Xgrade 3) in 9 patients (4 with BU/Mel, 5 with others). Veno-occlusive disease (VOD) occurred in two 1-year-old patients in the BU/Mel group. There were no treatment-related deaths. Of 4 (2 with BU/Mel, 2 with others) patients with central nervous system (CNS) relapse after HDC, 3 died. In conclusion, the BU/Mel regimen may be feasible for high-risk RB under careful monitoring for VOD, particularly in younger patients. CNS relapse associated with a lethal prognosis occurred after all regimens; therefore, further evaluation of HDC efficacy for high-risk RB is required.

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“…Rarely, heritable retinoblastoma is associated with an intracranial midline neuroblastic tumor, referred to as trilateral retinoblastoma (TRB). These tumors arise in the pineal and, less often, in the supra‐ or parasellar region and carry poor prognosis due to limited resectability, early leptomeningeal dissemination, and reduced sensitivity to common chemotherapeutic agents that can, however, be overcome by high‐dose chemotherapy (HCT) with autologous stem cell transplantation . Intracranial tumors in TRB have been classified as primitive neuroectodermal tumors, showing varying degrees of neuronal differentiation, but also exhibiting photoreceptor cell morphology, especially in cases of pinealoblastoma.…”

Section: Introductionmentioning

confidence: 99%

“…These tumors arise in the pineal and, less often, in the supra-or parasellar region and carry poor prognosis due to limited resectability, early leptomeningeal dissemination, and reduced sensitivity to common chemotherapeutic agents that can, however, be overcome by high-dose chemotherapy (HCT) with autologous stem cell transplantation. 3,4 Intracranial tumors in TRB have been classified as primitive neuroectodermal tumors, showing varying degrees of neuronal differentiation, but also exhibiting photoreceptor cell morphology, especially in cases of pinealoblastoma. It has, therefore, been proposed that they arise from primitive retinal progenitor cells dispersed along the subependymal layer anywhere along the cerebral midline.…”

Section: Introductionmentioning

confidence: 99%

Ectopic intracranial retinoblastoma in a 3.5‐month‐old infant without eye involvement and without evidence of heritability

Römer

Temming

Lohmann

et al. 2019

Pediatric Blood & Cancer

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Heritable retinoblastoma can rarely be associated with a midline intracranial neuroblastic tumor, referred to as trilateral retinoblastoma. We present an unusual midline brain tumor in an infant that was identified as ectopic retinoblastoma by histopathology, DNA methylation analysis, and molecular genetic detection of biallelic somatic inactivation of the RB1 gene. There was no ocular involvement, and germline mutation was excluded. In this nonresectable tumor, treatment with systemic chemotherapy including high‐dose therapy with autologous stem cell transplantation, but without definite local therapy, resulted in long‐lasting tumor control.

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High-dose chemotherapy followed by stem cell transplantation in the management of retinoblastoma: a systematic review

Cited by 17 publications

References 21 publications

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

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

High-dose chemotherapy for high-risk retinoblastoma: clinical course and outcome of 14 cases in the National Cancer Center, Japan

Ectopic intracranial retinoblastoma in a 3.5‐month‐old infant without eye involvement and without evidence of heritability

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