Robert P. Sundel, M.D.
Director, Samara Jan Turkel Clinical Center for Pediatric Autoimmune Disease

Thanks to the Samara Jan Turkel Foundation’s generosity and steadfast commitment, the Samara Jan Turkel Clinical Center for Pediatric Autoimmune Diseases is at the forefront of new advances in innovative patient care and cutting-edge research. Children’s Hospital Boston is truly grateful for your partnership in launching and sustaining this vital center. It is with deep gratitude that we share our achievements over the past year, made possible by your support.

We continue to make progress in building the awareness of the Center and its mission, so that we may reach more families in need of our comprehensive services.

Clinical Activities at the Samara Jan Turkel Center

Since the March 2003 report to the Foundation, approximately 50 additional patients have been treated at the Samara Jan Turkel Clinical Center.

The reputation of the Samara Jan Turkel Center continues to expand nationally. Families from Texas to Maine are being referred to the clinic—often having exhausted all possibilities in their local community. We are also responding to an increasing patient population in northern New England. Over the past year, the Center’s specialists have diagnosed extremely rare cases of autoimmune diseases, such as glomerulonephritis, dermatomyositis and CNS vasculitis. Exposure to these rare conditions advances the knowledge and expertise of our clinicians—allowing them to develop and share this knowledge with future patients and physicians.

The Center’s growing international reputation continues to bring us patients from around the globe. Examples of the countries represented by the Samara Jan Turkel Clinical Center include Mexico, Chile, Ecuador, and India. Oftentimes, patients are referred to Samara’s Center because we are on the forefront of research and therapeutic advancements—providing care that is only available to them at Children’s Hospital Boston. Patients come to Samara’s Center knowing they’ll receive expert diagnosis and novel therapies for a variety of conditions ranging from severe rheumatoid arthritis to vasculitis. Most of these conditions are difficult to diagnosis and often defy conventional treatments.

Enhancing Family-Centered Care

Caring for children struggling with autoimmune diseases extends far beyond skilled medical care. In keeping with the Children’s Hospital Boston’s philosophy, the Samara Jan Turkel Clinical Center is committed to treating the “whole” family—providing parents with the support services and educational resources needed to take an active part in their child’s care, both in the hospital and at home.

Many families who come to us are not aware of the organizations and support services available to them outside Children’s Hospital Boston. The Samara Jan Turkel Center serves as a critical link to much-needed resources for families struggling with their child’s chronic illness. Through the generosity of the Samara Jan Turkel Foundation, we’ve purchased important resources for parents such as educational books and training videos, and created parent and patient booklets focusing on autoimmune illnesses and conditions treated at our clinic.
Whether a patient is coming for a routine follow-up appointment, learning how to administer their injectable medicine, or coming in for the first time, any type of hospital visit can be an upsetting and stressful experience for a child. Being able to offer our patients fun, familiar rewards helps ease their fears. Samara’s Center continues to fund supplies for our treasure chests, so our young patients can choose “prizes” before leaving—putting smiles on their faces and ending their appointments on a positive note.
Although we make every effort possible to reduce waiting times when families come for outpatient appointments, some visits can last much longer than anticipated. For parents, it can be difficult to keep their children occupied while waiting to be seen by a physician. The Samara Jan Turkel Center tries to alleviate this stress by having toys, coloring books, and puzzles for children to use while waiting—an effort viewed as extremely helpful by parents.

Safety Net Programs

Many families cannot meet the unexpected and severe financials strains that arise when their child is chronically ill. As you know from our last report to the Foundation, support services provided by the Samara Jan Turkel Center include vital amenities such as hospital parking and meal vouchers, telephone cards, specialized medical equipment not covered by insurance, and financial assistance with transportation, babysitting services, groceries, and utility bills.

The following profiles highlight families in the Samara Jan Turkel Clinical Center who are benefiting from the Samara Jan Turkel Foundation’s generosity:

A five-year-old girl is treated at Samara’s Center for juvenile dermatomyositis—an autoimmune disease that causes severe muscle weakness and skin rash. As part of her treatment plan, she visits the Center for Ambulatory Treatment and Clinical Research weekly to receive pulse steroid infusions. These lengthy visits require her parents to take unpaid time off from work. The co-payments for these infusions can also be quite costly—putting a significant financial strain on the family. The Center helps offset their financial burdens by covering the costs of the co-payments. An excerpt from a thank you note written by the family illustrates the importance of this financial assistance:

“I am a recent receiver of your generosity through the Samara Jan Turkel Center for Pediatric Autoimmune Diseases at Children’s Hospital. In January, our 5-year-old daughter was diagnosed with dermatomyositis. Since then we visit Children’s Hospital weekly. Even with insurance, the costs are beyond our means. Assistance through your center was offered to me. We very much appreciate your help. The good news is we are told we will get our daughter’s health back completely in 2-3 years time. For that, I am sincerely grateful…”

A twelve-year-old boy with CNS vasculitis is admitted to Children’s Hospital every two weeks to receive treatment with cyclophosphamide, a chemotherapeutic agent. This treatment places a strain on both the patient and his family. It requires both parents to rearrange their work schedules and take unpaid days off to accommodate his treatment plan. When Children’s Hospital is particularly busy, we may lack bed space to accommodate even scheduled procedures. Thus, on one occasion due to unusually high volume, the family had to cancel the treatment on short notice and the mom had to take an extra day of work unpaid. Through the generosity of the Samara Jan Turkel Foundation, we were able to cover the cost of these lost wages for the family.
A twelve-year-old girl with polyarticular juvenile rheumatoid arthritis suffers from severe stiffness and poor range of motion, primarily in her hands and wrists. In extreme cases like hers, it is beneficial to use a paraffin unit as well as various forms of physical and occupational therapy. The paraffin units are not covered by MassHealth and can be financially impossible for many families to afford. Samara’s Center covered the cost of this unit to help improve joint mobility and muscle relaxation—enabling her to regain use of her fingers.
A seventeen-year-old female suffers from mixed connective tissue disease (MCTD) that is complicated by myositis. Her condition affects many parts of her body, especially the joints in her hands and wrists. In order for her to maintain proper oral hygiene, she needs to use an electric toothbrush—something neither her insurance company nor her family could provide. Through your funding, an electric toothbrush was purchased for her .
Samara’s Center continues to work with staff from many other departments to identify the needs of our patients not met by insurance companies. Children’s physical therapists, occupational therapists, and social workers alert us to children who need ring splints, orthotics, or other equipment they cannot afford. Thanks to Samara’s Center, we can help defray the costs of these expenses.

Clinical Research Highlights

The Children's Hospital Rheumatology Program, supported by the Samara Jan Turkel Center, is expanding the number of clinical trials of novel therapies for pediatric autoimmune diseases. Ongoing and planned protocols include:

Doppler ultrasonography for the evaluation of Juvenil Raynaud’s phenomenon (Role: Principal Investigator): Raynaud’s phenomenon is an exaggerated sensitivity to cold that leads to constriction of blood vessels and symptoms that may be debilitating upon exposure to low temperatures. At times, this is the initial manifestation of an autoimmune condition such as SLE or scleroderma. Using advanced ultrasound imaging, we are attempting to distinguish children with Raynaud’s phenomenon who are destined to develop a systemic autoimmune condition from those with benign sensitivity to cold.
Multicenter collaborative trial of corticosteroids in the initial treatment of Kawasaki Disease (Role: Center Co-PI): This is a large-scale NIH-funded project at 7 pediatric hospitals. The goal of this project is to confirm and expand earlier work, which suggests a beneficial role of corticosteroids in the treatment of Kawasaki disease.
Rituximab for the treatment of pediatric autoimmune conditions (Role: National protocol development): Rituximab, a biotechnologically generated monoclonal antibody directed against B-lymphocytes, is currently approved for the treatment of non-Hodgkin’s lymphoma. Since B-lymphocytes play a central role in the generation and propagation of autoimmunity, however, this drug also holds great promise for the treatment of lupus, dermatomyositis, vasculitis, and numerous other autoimmune conditions. Through the Samara Jan Turkel Center, we are taking a leading role in the development of protocols to evaluate this medication in the treatment of pediatric autoimmune conditions. Most recently, this included participation as the only pediatric autoimmunity and rheumatology center at a B-cell summit dedicated to the development of national treatment protocols.

Pioneering Basic Research

Children’s Division of Immunology is world-renowned as one of the largest pediatric research and clinical programs dedicated to improving the lives of children with immunologic and allergic diseases. The need for advances in identifying the molecular pathology of immunologic and allergic diseases is becoming more critical with dramatically improved diagnosis of immunodeficiency disorders, asthma, allergies, rheumatologic and autoimmune diseases.

As the nation’s first clinical center dedicated to autoimmune diseases in children, the Samara Jan Turkel Center strives to provide children with advanced diagnostic and genetic testing, and access to enrollment in clinical research trials for novel therapies. These efforts are performed in collaboration with the Division of Immunology’s basic research program. In the laboratories, scientific investigations are uncovering the molecular basis of immune disorders. By integrating biological and clinical research, we can speed the development of new treatments by translating laboratory findings to clinical practice as quickly as possible.

To further accelerate this pace of discovery, the Division of Immunology recently completed its move into Children’s new 12-story Research Tower. As the centerpiece of our $250 million Cause for Wonder campaign, this new facility increases Children’s overall research enterprise by 77%, thereby enabling important programs such as Immunology to recruit and retain both young investigators and elite scientists. With a faculty of over 30 physicians, researchers and postdoctoral fellows, the Division’s new Immunology Laboratories will be equipped with the most advanced technologies in an environment of cross-disciplinary collaboration—maximizing its potential in immunology research.

Thanks to the generosity of the Samara Jan Turkel Foundation, our laboratories will house the following equipment, which offers advanced analytical capability:

Immunoflourescence microscope: Significantly enhances our ability to view and examine cell substructures and track the movement of molecules. This microscope is crucial for diagnosing the source of inflammation in our patients and developing targeted treatment strategies.
Cary Spectrofluorometer package (including a cooler unit): Conducts sensitive fluorescence-based genetic and biochemical assays aimed at identifying the molecular basis of immunologic disorders. Since fluorescence-based assays are very sensitive, scientists can still analyze the blood cells in a sample even if only a small amount of the sample is available. This is particularly important since samples obtained from children, especially neonates, are often very small quantities [e.g. .5 ml (1/10 of a teaspoon) of blood drawn from a neonate as opposed to the 10 ml of blood typically drawn from an adult for diagnostic testing].
Spectrophotometer, Ultra cold freezer, and Deconvolution package (for the fluorescent microscope): The spectrophotometer is a vital instrument for various quantification purposes and will be used in many biochemical assays. The ultracold freezer allows us to store patient samples for future testing. Finally, the deconvolution package gives our scientists the ability to refine the images obtained by the fluorescent microscope and allows for the 3-dimensional reconstruction of images of sub-cellular structure. This equipment will drastically aid our study of pathology at the cellular level.

In recognition of your generosity supporting this important endeavor, Children’s Hospital and the Division of Immunology is honored to acknowledge the Samara Jan Turkel Foundation on a plaque outside the Division of Immunology’s fluorescent microassay laboratory.

Scientific Publications

Listed below are published research articles related to autoimmune conditions authored by faculty and staff of the Samara Jan Turkel Clinical Center within the past 12 months. Asterisks note publications that acknowledge the generosity of the Samara Jan Turkel Foundation.

Sundel RP , Update on the treatment of Kawasaki disease in childhood. Current Rheumatology Reports 2002; 4: 474-482
Fuhlbrigge RC, Nigrovic PA, Sundel RP. Raynaud's phenomenon in children: a retrospective review of 123 patients. Pediatrics 2003; 111: 715-721.
Sundel RP , Baker AL, Fulton DR, Newburger JW. Corticosteroids in the initial treatment of Kawasaki disease: Report of a Randomized Trial. Journal of Pediatrics 2003; 142: 611-616.
Baker AL, Bauvreau K, Newburger JW, Sundel RP, Fulton DR, Jenkins KJ. Physical and psychosocial health in children who have had Kawasaki disease. Pediatrics 2003; 111: 579-583.
Kocher M, Mandiga R, Murphy J, Goldmann D, Harper M, Sundel R, Ecklund K, Kasser J. A Clinical Practice Guideline for Treatment of Septic Arthritis in Children. The Journal of Bone and Joint Surgery 2003; 85: 994-999.
Sundel RP , Szer I. Vasculitis in childhood. Rheumatic Disease Clinics of North America 2002; 28: 625-654.
Binstadt BA, Caldas AM, Turvey SE, Weinstein HJ, Stone KD, Jackson J, Fuhlbrigge RC, Sundel RP. Rituximab therapy for multisystem autoimmune diseases in pediatric patients. Journal of Pediatrics 2003; 143: 598-604.

With deepest gratitude to the Samara Jan Turkel Foundation for its commitment to improving the lives of patients and families struggling with autoimmune diseases. So much has been accomplished through the Foundation’s partnership in establishing this model Center.

To sustain this momentum, we respectfully ask for your consideration of future support. Your continued generosity in helping the Center reach it’s next phase of growth and development will be recognized by The Samara Jan Turkel Foundation for autoimmune disease as well as Children’s Hospital Boston.

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