Treatment of WAS

Treatment of WAS - An Overview

Patients with WAS exhibit symptoms with varying degrees of severity. It's a complex medical condition affecting more than one system in the patient and is a challenging disease for parents and physicians to manage. It is therefore ideal to have a team approach in the care of these children. A basic team would consist of a pediatrician, an immunologist and a hematologist. The patient's regular needs can be taken care of by the pediatrician, who can coordinate the care with an immunologist and a hematologist. Transplant physicians are hematologists and may be willing to take care of these children even if they are not transplanted. Most of these children have eczema and can have skin disorders such as warts, molluscum, and herpes simplex. While most eczema can be effectively managed by the pediatrician, it is good to establish a relationship with a dermatologist.

Most WAS experts and many specialists understand the difficulty in finding physicians who are knowledgeable in the care of these patients and will continue to care for them into adulthood. Taking the time to build a strong and trusting relationship with physicians goes a long way in the care of these children. Having clear guidelines set out by the physician can ease stress and anxiety and provide children a framework within which they can grow. As the children get older, they can communicate directly with the physician who will be able to set aside his or her emotions and will be able to help them with compassion, understanding and authority. 

During each of these visits, it is helpful to have the results of all the tests, labs and meeting summaries (consult notes) filed by date. Please request the consulting physicians i.e. the immunologists and hematologists to forward consult notes to the primary care physician. Please request WAS experts to forward their consult notes to the specialists and the primary care physician. This improves communication between the various physicians resulting in better patient care.

The Role of the WAS Expert: Wiskott-Aldrich Syndrome is a complex and dynamic disorder with varying presentations that continue to baffle even the experts who have been working with these patients for decades. In many cases, there is a lack of consensus on management of the disease. These experts have a tremendous knowledge base and experience about the varied presentations, the genotypes and phenotypes, the treatment options that are most suitable and information about recommended transplant centers. They are used to talking with parents who are in difficult positions and can soothe the most frazzled of parents. In the rapidly evolving world of medicine, the WAS experts can guide immunologists, hematologists and parents on the best possible option for each patient. The WAS experts can provide a one time consultation or can be available to consult on a regular or as needed basis. These experts fully understand the difficult position that the physicians and parents are in and are most helpful and accommodating. The following situations are the ones that experts are most often consulted in:

• Physicians who have a newly diagnosed patient with WAS and would like to know the best advice to give the parents.

• Help with interpretation of typical clinical findings and diagnosis of complications of WAS.

• Counseling on establishing patient-specific management plan, based on disease severity evaluation and available therapeutic options.

Physicians and parents may choose different treatment options according to the severity of the diseases, but at this time there is no consensus on a consistent method of management for all cases. Outlined are the three different options that are available to parents.  A hematopoietic cell transplant is the only proven cure for WAS. Since 2007, gene therapy has been shown some encouraging results on several patients with no major untoward effects. Even though most patients are transplanted there are a significant number of patients, particularly the milder cases who are being managed conservatively.

For more information visit these sections:

Hematopoietic Cell Transplant

Preventative and Vigilant Care

Gene Therapy

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