Outcomes following autologous hematopoietic stem cell transplant for patients with relapsed Wilms’ tumor: a CIBMTR retrospective analysis

Abstract

Despite the dramatic improvement in the overall survival for patients diagnosed with Wilms’ tumor (WT), the outcomes for those that experience relapse have remained disappointing. We describe the outcomes of 253 patients with relapsed WT who received high-dose chemotherapy (HDT) followed by autologous hematopoietic stem cell transplant (HCT) between 1990 and 2013, and reported to the Center for International Blood and Marrow Transplantation Research (CIBMTR). The 5-year estimates for event free survival (EFS) and overall survival (OS) were 36% (95% CI; 29 – 43%) and 45% (95% CI; 38 – 51%) respectively. Relapse of primary disease was the cause of death in 81% of the population. EFS, OS, relapse and transplant-related mortality (TRM) showed no significant differences when broken down by disease status at transplant, time from diagnosis to transplant, year of transplant or conditioning regimen. Our data suggest that HDT followed by autologous HCT for relapsed WT is well tolerated and outcomes are similar to those reported in the literature. Since attempts to conduct a randomized trial comparing maintenance chemotherapy with consolidation versus high-dose chemotherapy followed by stem cell transplant have failed, one should balance the potential benefits with the yet unknown long-term risks. Since disease recurrence continues to be the most common cause of death, future research should focus on the development of consolidation therapies for those patients achieving complete response to therapy.

High dose chemotherapy and autologous hematopoietic cell transplantation for Wilms tumor: a study of the European Society for Blood and Marrow Transplantation

“…The proportion of patients transplanted for recurrent WT seems to have increased over time, [18,19] although randomized comparison of the potential additional benefit of such an approach over systematically intensifying nonmyeloablative chemotherapy has never been investigated. We know that evidence for efficacy of HDT with aHCT in patients experiencing relapse after treatment for their WT is limited.…”

“…Since the first European Blood and Marrow Transplant (EBMT) report, [18] the number of transplanted patients with WT included in the EBMT registry has grown to over 400 cases, most of them treated outside clinically controlled protocols. In a recent analysis from the Center for International Blood and Marrow Transplantation Research, outcomes on 253 children with relapsed WT have been described [19].…”

High‐dose chemotherapy without whole lung radiation for refractory pulmonary metastases in an infant with stage IV favorable histology Wilms tumor

“…Although the safety and efficacy of HDC for recurrent and refractory WT are not completely evaluated, high-risk patients may benefit from HDC. [7][8][9] Achieving continuous remission without WLR for patients with FHWT with pulmonary metastases is worthwhile. With HDC at CR, our case has remained in continuous remission without WLR.…”

“…Of these 13 patients, 1 patient with pulmonary relapse achieved CR following salvage chemotherapy and remained disease‐free without WLR. Although the safety and efficacy of HDC for recurrent and refractory WT are not completely evaluated, high‐risk patients may benefit from HDC …”

Mucosite oral em pacientes pediátricos submetidos a transplante de células hematopoiéticas

“…A principal falha no tratamento é a recidiva da neoplasia primária (Dalle et al, 2018). Contudo, a recorrência da doença continua a ser a causa mais comum de morte para esses pacientes (Malogolowkin et al, 2017).…”