We recently initiated a series of invited articles on subjects of international importance in pediatric hematology and oncology. This issue includes the first two of this series. Dr. Finlay reviews progress in the treatment of childhood brain tumors and what we can expect in the near future. Overall, the childhood brain cancer 5-year survival rate has increased from 58% (1975–1979) to 73% in the period from 1995 to 2000. Advanced imaging, sophisticated neurosurgical techniques, and improved chemotherapy and radiation protocols have each played a part in this improvement. However, increased morbidity from these interventions, including “posterior fossa” syndrome, has resulted. And whereas the 5-year survival rate for average risk medulloblastoma has increased from 56% to 90%, problems of inadequate staging, standardization of surgical techniques, and long-term sequelae remain significant obstacles to overcome. Studies are in progress comparing full posterior fossa irradiation with focal conformal irradiation to the tumor bed, as well as studies comparing lower radiation doses in younger children.
Dr. Borgna-Pignatti summarizes the progress made in treating thalassemia major. In a natural history study following over 1000 patients, a dramatic decrease in mortality can be seen in the younger patients. In a subcohort of over 700 patients born after 1970, heart failure was present in only 6% of the population. Survival and complications directly correlated with patient iron stores and serum ferritin. Approaches to improving iron balance with combination therapy are reviewed, as well as the availability of new oral iron chelators. Previously unrecognized medical complications are now increasing in the older population, including pulmonary hypertension, thrombosis, and pseudoxanthoma elasticum. Finally, the enormous financial burden of care is summarized.
We would be delighted to have feedback from our readers as to the benefit of these invited articles and suggestions for future topics.
Finally, we would like to make researchers aware of a request for applications for 2007–2008 grants in translational research in adult thallassemia from the Cooley's Anemia Foundation. The goal of this initiative is to recruit investigators from all disciplines and backgrounds (MD, RN, PhD, MPH, MSW) to address one or more of the following areas in terms of the adult patient with thalassemia: fertility, pregnancy and family planning; quality of life/psychosocial; burden of disease; the heart; and gene therapy. Please visit the web site at www.cooleysanemia.org, contact Cooley's Anemia Foundation at [email protected], or myself at [email protected] for more information.