Caring for children and young people is always challenging, however experienced the clinician and however limitless the budget. Skillful management demands a prompt, accurate diagnosis, careful explanation of potential therapies, and information about prognosis. Parents and children need reassurance that they will be cared for indefinitely by a team experienced in the management of pulmonary hypertension. When pulmonary hypertension (PH) is a complication of a recognised disease process, such as congenital heart disease or connective tissue disease, then the parents and children are anxious about the new, additional problem. When a child who was previously thought healthy and entirely normal is found to have an incurable disease, the shock is almost intolerable. All children with pulmonary hypertension need specialised care and should only be treated in ‘centres of excellence’.
Some of us are fortunate enough to be able to offer any therapy required, including lung transplantation, but most physicians are not so fortunate. This is why the PVRI International Guidelines for Patient Management need to be modified for use in the developing world. Professors Ghazwan Butrous and Majdy Idrees have worked hard on producing guidelines for the developing world. Each region or country can amend these guidelines to suit their own needs and resources, and distribute the guidelines in their own language.
In managing any patient with PH the diagnostic algorithm is the same, but the diagnosis is frequently more complicated in children than adults. All the causes of pulmonary vascular disease (PVD) found in adults are seen in children, but children have many additional problems. The development or early maturation of the pulmonary vasculature may be abnormal. The child may be syndromic, when the chromosomal abnormality may or may not have a recognised association with PH. To counter these issues, the PVRI Paediatric Task Force, led by Dr Ian Adatia, produced a new Disease Classification of Pulmonary Vascular Disease in Childhood at the Annual Congress in Panama in 2011. In reality, the long list of possibilities is a box-ticking exercise which ensures that the phenotype is accurately and comprehensively described. In addition, it can be a struggle to describe functional capacity in children using the WHO classification. The 6-minute crawl test doesn’t work! Therefore we worked out a more precise but simple classification of functional ability in children by comparing their performance with that expected in a normal child of the same age: the PVRI Panama Functional Classification.
Paediatric trials are intrinsically complicated, since issues of consent and ethics and long term pharmacovigilance are difficult. New therapies are slow to come to the market, whilst medicines are often expensive and do not provide a cure. Nevertheless, we have made a huge amount of progress in the treatment of children during the last fifteen years and we must persist.
By focusing attention on the needs of children with pulmonary vascular disease the PVRI can and should play a significant role in improving the outlook for these patients.