The Project

As childhood mortality related to communicable disorders improves, cancer emerges as a major cause of death and disability in developing countries, where health systems are often poorly equipped to deal with this challenge.

Despite numerous obstacles, pediatric malignancies can be effectively treated and cured in resource-limited settings. Although the outcome of patients with high risk, stage IV NB remains poor, even in developed countries, with an estimated 8-year even-free survival (EFS) of 44%, the steady improvement in the intermediate risk group patients is promising, with > 85% 3-year EFS. Numerous clinical trials demonstrated that surgery alone for low risk patients (Stage I and II), was sufficient to maintain an excellent 3-year EFS at >95%. There are wide differences in approach to treatment strategies at medical centers globally.

There have been significant limitations in these reports due to lack of clear data on extent of resection and secondary non-life threatening surgical complications. These reports also have not delineated response and treatment outcomes based an age parameters, thereby limiting critical comparisons. The approach to treatment in patients in low-, and middle-income countries (LMIC) with Stage II and III NB has involved significant variation over the course of last three decades. The limitations of the current reports are that these span a period extending as great as 35 years with significant inter-institution treatment variability. The outcome measures are inconsistently monitored over a given length of time.

One of the biggest challenges of centers in developing countries is their ability to diagnose and stage NB due to inadequate tissue procurement, unavailability of radiographic studies such as MIBG and CT scans. Other critical challenges may prevent risk-stratification of patients based on tumor genetic and molecular profiles. Outcome for patients with high risk, Stage IV NB is uniformly poor worldwide. Treatment variability for high risk patients with stage IV NB does not appear to have altered the poor outcome seen in many of the developing countries. The lack of infrastructure and access to medications to provide the intensive therapy required to target this patient population may be one of the most critical limitations for the poorer outcome. Ongoing education, needs assessment and assistance via cooperative development of treatment strategies and effective supportive care has been shown to improve outcome of children with cancer globally. NB, however, given its low curability in high-risk patients, which represent over 50% of cases, has lagged behind.

Project leaders are among the prime neuroblastoma experts and include highly reputed institutions such as University of California at San Francisco (Dr Kate Matthay), St. Jude’s Children’s Research Hospital (Drs Scott Howard and Catherine Lam) and Connecticut Children’s Medical Center (Dr Nehal Parikh). This initiative is connected to the International Society of Pediatric Oncology in Developing Countries (SIOP-PODC) currently led by Dr Scott Howard, and builds on the experience of the Cure2Children Foundation (www.cure2children.org), an Italian organization which has successfully supported the development of tertiary care programs for pediatric cancer and severe blood disorders in LMIC.