Overall survival for neuroblastoma in South Africa between 2000 and 2014

Heerden, Jaques van; Hendricks, Marc; Geel, Jennifer; Sartorius, Benn; Hadley, G. P.; Plessis, Jan du; Büchner, A.; Naidu, Gita; Emmenes, Barry van; Zyl, Anel van; Kruger, Mariana

doi:10.1002/pbc.27944

Cited by 23 publications

(32 citation statements)

References 39 publications

(64 reference statements)

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“…Heterogeneous resource allocation, with dependence on private insurance, restricted the availability of ASCT to 3.7% HR‐NB patients in South Africa, with radiotherapy and cis‐retinoic acid also having limited availability. The 2‐year countrywide OS in South Africa for HR‐NB patients was 27.6% 31 . Additionally, merely one patient was lost to follow up in our cohort after the treatment plan was decided.…”

Section: Discussionmentioning

confidence: 93%

Autologous stem cell transplant for high‐risk neuroblastoma: Achieving cure with low‐cost adaptations

Jain¹,

Hans

Totadri³

et al. 2020

Pediatric Blood & Cancer

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Background:The majority of patients in low-and middle-income countries (LMIC) are unable to receive optimal therapy, including autologous stem cell transplant (ASCT) for high-risk neuroblastoma. Management is intensive and multidisciplinary; survival is often poor. We report a singlecenter outcome of high-risk neuroblastoma, with adaptations optimized for LMIC. Procedure:The study was retrospective. Patients were treated on the backbone of the highrisk neuroblastoma study-1 of SIOP-Europe (HR-NBL1/SIOPEN) protocol with ASCT. Adaptations incorporated to decrease cost, requirement for inpatient admission, infections, and faster engraftment included (a) optional outpatient administration for rapid-COJEC, (b) two sessions of stemcell apheresis, (c) storing stem cells at 2-6 • C without cryopreservation for up to 7 days, (d) no central lines, (e) no antibacterial/antifungal/antiviral prophylaxis, (f) omitting formal assessment of cardiac/renal/pulmonary functions before ASCT, and (g) administration of pegylated granulocyte colony-stimulating factor on Day +4.Results: Over 5 years 9 months, 35 patients with high-risk neuroblastoma were treated. Rapid-COJEC was administered over a median duration of 80 days (interquartile range: 77, 83). Conditioning regimen included melphalan (n = 7), oral busulfan-melphalan (Bu/Mel; n = 6), or intravenous Bu/Mel (n = 22). The median viability of stem cells stored for 6 days (n = 28) was 93% (range: 88-99). Two (5.7%) patients had ASCT-related mortality. The 3-year overall and event-free

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Section: Discussionmentioning

confidence: 93%

Autologous stem cell transplant for high‐risk neuroblastoma: Achieving cure with low‐cost adaptations

Jain¹,

Hans

Totadri³

et al. 2020

Pediatric Blood & Cancer

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“…Among 295 patients with HR-NB in the National data from South Africa, merely 11 (3.7%) received ASCT due to limited availability and dependence on private insurance. 18 Similarly, 8.8% of patients with stage 4 neuroblastoma were consolidated with ASCT in a single-center report from New Delhi, India. 19 Selected reports on the survival of HR-NB without ASCT and dinutuximab are summarized in Table 3.…”

Section: Discussionmentioning

confidence: 99%

“…A relapse or progressive disease was noted in 11 (39%) patients at a median duration of 17 months (IQR: 5,18). Following the completion of therapy, 2 (7%) patients died at home at 21 and 31 months of follow up.…”

Section: Survivalmentioning

confidence: 99%

Survival in patients with High-Risk Neuroblastoma without autologous stem cell transplant and dinutuximab

Jain¹,

Trehan²,

Menon³

et al. 2020

Preprint

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Background: The majority of patients with high-risk neuroblastoma (HR-NB) in low-and middle-income countries (LMIC) do not have access to autologous stem cell transplant (ASCT) and dinutuximab. Consolidation with non-myeloablative chemotherapy is not well-defined, and the outcomes are variable. We report a single-center outcome of patients with HR-NB, treated with non-myeloablative consolidation. A tabulated compilation of similar reports is included. Procedure: A retrospective chart review of patients with HR-NB was performed from January 2009 till June 2016. Patients were treated on the backbone of HR-NBL1/SIOPEN protocol. Treatment included induction with rapid-COJEC, surgery, consolidation, radiotherapy to the primary tumor, and differentiation therapy with isotretinoin. Consolidation included 4 cycles of topotecan, vincristine, and doxorubicin (TVD) instead of ASCT. Infusion of vincristine and doxorubicin were modified for ease and to enable administration in daycare. Results: Over 7-½ years, 28 patients with HR-NB were treated. Two (7%) patients had therapy-related mortality. A relapse or disease progression occurred in 11 (39%) patients at a median duration of 17 months (IQR: 5, 18). Treatment abandonment was observed in 4 (14%) patients. The 4-year event-free survival was 29.3%. The median follow up of disease-free patients is 49 months (IQR: 45, 79). Patients with relapse were not treated further. Conclusions: A 4-year EFS of 29.3% was observed when 4-cycles of TVD were administered instead of ASCT in patients with HR-NB. The study and the review will aid stakeholders in LMIC for decision-making while considering the options of treatment for HR-NB if access to ACST and dinutuximab is lacking.

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“… 20 , 21 This was achieved by applying ATRs formulated by the SIOP-PODC-ATR group in a stepwise manner, with increased research capacity and survival outputs as priorities of the trials. 24 , 36 , 59 Similarly, clinician-researchers in South Africa have developed multicenter national clinical trials to improve outcomes of patients with Hodgkin lymphoma, 60 neuroblastoma, 61 and germ cell tumors. 62 The development of a limited number of treatment protocols using the ATR framework to create regimens suitable for particular settings will both improve data quality and allow increased reproducibility and validation of study data.…”

Section: Discussionmentioning

confidence: 99%

Pediatric Oncology Clinical Trials and Collaborative Research in Africa: Current Landscape and Future Perspectives

Heerden

Zaghloul

Neven

et al. 2020

JCO Global Oncology

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PURPOSE Adequate clinical services have yet to be established in the majority of African countries, where childhood cancer survival rates vary from 8.1% to 30.3%. The aim of this review is to describe the landscape of pediatric oncology trials in Africa, identify challenges, and offer future opportunities for research collaborations. METHODS The study includes data from the International Pediatric Oncology Society (SIOP) global mapping survey, meta-research identifying trials in Africa in ClinicalTrials.gov, and a literature overview of publications on the subject of pediatric oncology clinical research supported by expert opinions on the current situation and challenges. RESULTS The SIOP global mapping survey received responses from 47 of 54 African countries, of which 23 have active clinical research programs. A preliminary search of ClinicalTrials.gov showed that only 105 (12.1%) of 868 African oncology studies included children and adolescents. Of these, 53 (50.5%) were interventional trials according to the registry’s classification. The small number of African trials for children and adolescents included palliative care and leukemia trials. In African oncology journals and international pediatric oncology journals, < 1% of the pediatric oncology publications come from Africa. Services and research were strengthened by international collaboration. National studies focused on clinical needs, local challenges, or interventional priorities. Both the literature review and the expert opinions highlight the need to expand clinical research in Africa, despite ongoing regional instability and lack of resources. CONCLUSION While a low number of pediatric clinical treatment trials are open to African children and adolescents, clinical research of high quality is being done in Africa. Several initiatives are stimulating the development of the research capacity across the continent, which should increase the publication output.

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Overall survival for neuroblastoma in South Africa between 2000 and 2014

Cited by 23 publications

References 39 publications

Autologous stem cell transplant for high‐risk neuroblastoma: Achieving cure with low‐cost adaptations

Autologous stem cell transplant for high‐risk neuroblastoma: Achieving cure with low‐cost adaptations

Survival in patients with High-Risk Neuroblastoma without autologous stem cell transplant and dinutuximab

Pediatric Oncology Clinical Trials and Collaborative Research in Africa: Current Landscape and Future Perspectives

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