Part I General problems
1 The size and nature of the problem
John A. Dodge
Thirty years ago it was not uncommon to find groups of small children scaring each other with tales of pneumonia, diphtheria, or tuberculosis. Similarly, a century earlier cholera and scarlet fever were childhood bogies. Today’s children are scarcely aware of such diseases, and certainly their existence presents them with no threat. Social development and advances in medical knowledge since the mid-nineteenth century have virtually eliminated these sources of fear. Equally, the sweeping social changes of the last century, and the improvements in living standards and domestic hygiene, have meant that it is no longer a matter for favourable comment when a mother of a large family successfully rears all her children: this is what we have come to expect. Now, not only the fittest survive, but death, when it comes to a child, is the more bitter for being stealthy, unexpected and selective.
Today, childhood deaths are relatively infrequent (Table 1 summarises details for the United Kingdom), the most numerous being in the immediate newborn period, from disorders associated with prematurity and the birth process. Infant deaths are often sudden and unexplained (‘cot death’), and throughout childhood accidental death figures prominently in the mortality statistics. Such children die after a very short illness, or none at all, and are not therefore the subjects of this chapter.
Table 1 England and Wales 1970* Deaths, all causes 0—14 years | 18,997 |
Tuberculosis | 26 |
Poliomyelitis | 5 |
Diphtheria | 0 |
Neoplasms (including leukaemia) | 844 |
Respiratory diseases | 3,210 |
Cystic fibrosis | 132 |
Accidents, poisoning and violence | 2,042 |
Congenital anomalies | 3,526 |
*From the Registrar General’s Report.
In contrast to the last century, death from infections such as pneumonia is uncommon, unless it occurs in a child who is already chronically ill or congenitally malformed, when it is simply a terminal event. Malnutrition—a major underlying factor which predisposes children to die with an infection—has largely been removed from our community, though its presence in developing countries contributes to high mortality rates and continues to cause concern.
Despite all this, from time to time the paediatrician, surgeon or family doctor finds himself telling parents that their child has a deadly disease. It may be a malignant process, such as leukaemia or a brain tumour, or it may be an inherited progressive disorder of the nervous system or muscles. Other conditions such as cystic fibrosis have a variable but not inevitably downward course, so that the prognosis, although statistically gloomy, must be given very cautiously. The fact is that we can rarely predict with any degree of accuracy the duration, or severity of course, which any individual child’s illness will take. Nevertheless, analysis of records from different centres enables us to obtain a clear picture of average survival times from diagnosis, and current trends towards more effective treatment.
Childhood malignancy
In the field of childhood malignancy, tumours of the brain are the most numerous (Table 2). They are of various types and found in several different sites, and although all present problems of diagnosis and treatment, some carry a relatively good outlook. The onset is usually insidious. Fits, a common presenting symptom of cerebral tumours in adults, are much less frequent, and occur later, when a child is the victim. This is probably due to the fact that most childhood tumours lie in the brainstem, whereas in adults the majority of growths occur in the cerebral hemispheres. The occurrence of persistent headaches, often accompanied by vomiting, may indicate that the intra-cranial pressure is raised, but occurs whether the cause is a tumour, an abscess, meningitis or hydrocephalus, and is in no way specific. Highly malignant tumours of the brainstem are inoperable and rapidly fatal, with sometimes a limited response to radiotherapy. Tumours in the cerebellum may disturb balance and limb movements, but are sometimes slowly growing, relatively benign, and susceptible to complete removal leaving varying degrees of handicap. An unusual variety of tumour, the craniopharyngioma, arises from an embryological remnant in the region of the pituitary gland, and by local pressure produces impairment of the hormonal regulation of growth and sexual development, as well as interfering with vision. Although it is not usually malignant, it can be extremely difficult to remove because its situation is almost inaccessible without serious damage to vital structures. Pineal gland tumours are rare, but may present as precocious puberty, also due to a disturbance of the endocrine system.
Table 2 Approximate annual incidence of certain childhood illnesses in Great Britain* Cerebral tumours | 700 |
Other tumours (kidney, adrenal, embryonic, etc.) | 600 |
Leukaemia, lymphatic | 320 |
Leukaemia, myeloid | 80 |
Cystic fibrosis | 600 |
Degenerative diseases of the brain and nervous system | <100 |
Measles | 300,000 |
Spina bifida | 3,000 |
*Sources: Registrar General’s Survey, 1967-71 and personal communications from the Cystic Fibrosis Research Trust, the Leukaemia Society, and the Hospital for Sick Children. Great Ormond Street. London.
With the exception of some tumours which are associated with rare inherited disorders of the nervous system and skin (neurofibromatosis, tuberose sclerosis), the aetiology of brain tumours is quite unknown. The prognosis is bad, death usually occurring within months of diagnosis in the case of brainstem tumours. In the more benign, well defined tumours of the cerebellum or cerebral hemispheres, cystic changes may occur and even when complete surgical removal is not possible a fatal outcome may sometimes be postponed for many years by simple drainage. Similarly, relief of pressure can sometimes be brought about by inserting a drainage tube into the ventricles of the brain with the other end passing into the circulation. In this way worthwhile palliation may be provided particularly when a slowly growing tumour is situated in such a way that it obstructs the flow of cerebro-spinal fluid. It must be emphasised, however, that the brain tumours most frequently encountered in children are not really amenable to any of the treatments currently available.
Near to the brain, the retinoblastoma is a highly malignant tumour of the eye. It has an hereditary basis. Removal of the eye may be curative, along with drug treatment. When, as sometimes occurs, the tumours are bilateral, the more severely affected eye is removed and a suitable drug can be injected into the artery supplying the other. Like most very malignant tumours, it is very sensitive to radiation and to chemical agents, so that in the absence of widespread secondary deposits a favourable outcome may follow if the tumour has been detected in time.
Abdominal tumours are next in order of frequency to those of the brain. Within the abdomen, neuroblastomas arising from the adrenal glands are slightly more common than nephroblastomas arising from the kidneys. Both of these tumours are only found during childhood. The neuroblastoma is actually derived from the adrenal medulla, the part of the gland which originated in the embryo from the primitive nervous system. Neuroblastomas are very malignant, and tend to produce secondary deposits at distant sites such as the long bones of the limbs, and, characteristically, in the orbit behind the eye. They are unusual tumours in that they occasionally undergo spontaneous regression, so that evaluation of treatment is not always easy. It was this fact which led to a vogue for treating neuroblastomas with injections of vitamin B12 but later trials failed to confirm its usefulness. With a combination of agressive surgery and radiotherapy, three-year survival rates of up to 60 per cent are being reported, and a large proportion of these children will probably be completely cured. Neuroblastomas of infancy have a decidedly better prognosis than those occurring in older children. Following appropriate treatment, it is possible to test the urine at intervals for specific products of the tumour, and by this means recurrences can be detected.
Nephroblastomas are also known as Wilm’s tumours. They occur in the kidney and may come to light when the child passes blood in his urine, or on feeling a large hard mass in his abdomen. In recent years treatment has been directed along several lines: complete surgical excision of the tumour followed by radiotherapy. Distant secondary deposits often respond to chemotherapy with agents such as actinomycin D, and sometimes surgical removal of secondaries is also worthwhile.
Current opinion is that whenever possible treatment of childhood malignancies should be centralised on a regional basis, so that an experienced team of physicians, surgeons, radiotherapists and pathologists may bring to each young patient the necessary expertise.
The same requirements obtain for leukaemias which are essentially a form of malignancy which affects the white blood cells. The peak incidence in children is between the ages of two and four years. The most common variety is acute lymphatic leukaemia where microscopic examination of a blood film characteristically shows a greatly enlarged number of primitive lymphocytes. The lymphatic glands of the ...