Aplastic anemia program

Aplastic anemia is a serious bone marrow disease, but it’s also rare. While other pediatric hospitals may see just one or two aplastic anemia patients in a year, Children’s Wisconsin treats these children every day.

That’s because, more than 50 years ago, we developed the standard of care for aplastic anemia. In 1977, Bruce Camitta, MD, who arrived at Children’s Wisconsin just a few years later, wrote Camitta Criteria for aplastic anemia. Two decades later, Dr. Camitta passed the baton to David Margolis, MD, a nationally renowned aplastic anemia expert who serves as Pediatrician-in-Chief at Children’s Wisconsin.

“We have a long-standing history of being leaders in the area of aplastic anemia,” says Dr. Margolis, a medical advisory board member for the Aplastic Anemia and MDS International Foundation. “Thanks to the way Dr. Camitta set up the program, we manage each case individually through a lens of experience.”

The Children’s Wisconsin difference

From diagnosis onward, every aplastic anemia patient at Children’s Wisconsin is treated by our bone marrow transplant team whether the child eventually has a transplant or not. The reason: our bone marrow transplant team is the most experienced with the medications and treatments used for aplastic anemia, whether it’s a bone marrow transplant or immunosuppressive therapy. We believe in building long-term relationships with our patients. So, if a child eventually needs a bone marrow transplant, familiar faces will guide them through the process.

Located in the MACC Fund Center for Cancer and Blood Disorders at Children’s Wisconsin, our dedicated aplastic anemia team includes:

• Board-certified physicians David Margolis, MD, and Larisa Broglie, MD, MS
• Four advanced practice providers: Lynnette Anderson, Elizabeth Thieleke, Miranda Gries and Kellyn Munson
• Two nurse clinicians
• Social workers
• Psychologists
• Child life specialists
• A dietitian
• Art and music therapists

Our aplastic anemia providers are experienced with the intricacies of this rare disease, and they know there are multiple ways for a child to get better. That’s why we take an individualized approach to caring for each patient, thoroughly getting to know the children and their families. We walk through each treatment option to determine the best fit for every patient and family.

Our tight-knit team can move quickly and seamlessly between various treatments. We provide exceptionally close patient monitoring, as well as easy access. We’re always responsive—a provider is on call around the clock.

What we offer

Children’s Wisconsin provides comprehensive aplastic anemia care from diagnosis to post-treatment and beyond.  We work with other experts in aplastic anemia throughout the country and internationally.  We are part of the North American Aplastic Anemia Consortium is a collaboration of aplastic anemia experts whose goal is to develop better therapies for children with aplastic anemia.  We also collaborate with the PTCTC and BMT-CTN to be at the forefront of clinical research trials for children needing blood and marrow transplant.

Diagnosis: Many patients first come to us with unexplained low blood counts or symptoms that may include bleeding, bruising and fevers. We begin by performing a bone marrow biopsy to better understand the issue and rule out other diseases. If aplastic anemia is diagnosed, we’ll determine if the condition was inherited (developed because of a genetic predisposition) or acquired (occurred without a known predisposing illness), which can help determine the patient’s treatment.

Second opinions: Children and families who received aplastic anemia diagnoses at other medical centers can consult our doctors on treatment options.

Bone marrow transplant consultation: Our team can consult with patients who received immunosuppressive therapy elsewhere and are considering a bone marrow transplant.

Complications: Patients who experience complications after a bone marrow transplant can come to Children’s Wisconsin to discuss alternative treatment options.

Treatment

The most common and effective treatments for aplastic anemia are bone marrow transplants from related or unrelated donors and immunosuppressive therapy. Our aplastic anemia team has guided patients through these options countless times, so patients and families can feel confident in the treatment choices they make.

Bone marrow transplant—matched donor: We’ll facilitate the entire process, from determining whether a patient has a related donor (sibling or other haploidentical related donor) to the post-transplant care.

Bone marrow transplant—alternative donors - unrelated donors and haploidentical related donors: If a donation from a sibling or other related donor is unavailable, a bone marrow transplant from an unrelated donor may be an option for some aplastic anemia patients. We’ll search national and international bone marrow registries for donors with close tissue matches. Outcomes for these transplants have drastically improved in recent decades, nearly approaching the high success rates for related donor transplants.

Immunosuppressive therapy: When bone marrow transplant isn’t an option—or isn’t the best option—we offer immunosuppressive therapy to our aplastic anemia patients. These medications, which reduce the body’s immune system activity and can treat bone marrow failure, are administered intravenously in the hospital.

Survivorship

Our aplastic anemia team at Children’s Wisconsin follows patients long after their bone marrow transplants or immunosuppressive therapy is complete. Depending on the treatment, we’ll see patients for follow-up appointments semi-weekly, weekly, monthly and, eventually, yearly.

Children’s Wisconsin also offers a survivorship program for patients who have finished their aplastic anemia treatment. Along with tracking patients for potential late effects of their treatment, our Next Steps Survivorship Program offers education, resources, community and ongoing support to our patients.

Our pediatric aplastic anemia patients have gone on to graduate from high school and college, get married, start families and even become nurses and other medical professionals. Our patients—past and present—continue to inspire us every day.

Call (414) 266-2420 to schedule an appointment or to learn more about our services.