Bone Marrow Failure Syndromes

Bone marrow failure syndromes are a group of conditions in which blood-producing cells inside the bones don’t function as they should. The best care includes advanced therapies from specialists who regularly treat these conditions. You’ll find these offerings along with patient- and family-centered support at Children’s Hospital Los Angeles.

Our pediatric hematologists are leaders in treating bone marrow failure syndromes. Your child has access to innovative therapies, some of which are available only in select programs worldwide. We give hope to children for whom previous treatments weren’t successful.

Care for Aplastic Anemia and Other Bone Marrow Failure Syndromes: Why Choose Us

Our team includes internationally recognized pediatric hematology specialists. We coordinate the services that best meet your child’s needs to help achieve outstanding results.

As part of CHLA’s internationally recognized Hematology program, our bone marrow failure syndrome offerings include:

  • Highly skilled team: Bone marrow failure is rare, but our hematologists excel in treating it. We offer decades of experience, and our team is finding new care methods through clinical trials. Our specialists give your child the best available therapies. Meet our team.
  • Bone marrow transplant excellence: Transplants are a common treatment for bone marrow failure. At CHLA, your child is in the hands of experts. Our commitment to safety and the highest transplant standards has earned our program national recognition. Learn more about our Transplantation and Cellular Therapy Center. 
  • Innovative treatments: We successfully deliver bone marrow transplants including using half-match (haploidentical) transplants. This option uses bone marrow from blood relatives. And our research is leading the way in using unrelated-donor marrow. These techniques are not widely available and give your child more options for treatment.
  • Clinical trials: Our team participates in clinical trial networks exploring the next generation of bone marrow failure syndrome treatments. Your child may be eligible to receive new therapies designed to be gentler on the body and have a lower relapse risk.

What Is Bone Marrow Failure?

Bone marrow is the spongy substance inside certain bones where blood-producing cells form. Bone marrow failure occurs when the body doesn’t produce enough of these cells or they cannot do their job. Children with these conditions can become sick again (relapse) even if initial treatments are successful.

Conditions (syndromes) that can lead to bone marrow failure include:

  • Aplastic anemia
  • Paroxysmal nocturnal hemoglobinuria
  • Myelodysplastic syndrome (MDS)
  • Thrombocytopenia

Patient- and Family-Focused Support for Children With Bone Marrow Failure

Our support services make living with bone marrow failure a little less stressful on your child and family.

Services include:

  • Child Life specialists: Using age-appropriate methods, we explain tests and treatments so your child knows what to expect. Specialists also help your child cope with anxiety before, during and after treatments. Read more about our Child Life Program.
  • Language and communication services: If English is not your first language, qualified interpreters and translators can help you communicate with your child’s care team. Get more information about Interpreting and Translation Services.
  • Transition program: We help prepare young adults to smoothly transition to adult blood disease specialists. Using a team approach, we teach adolescents how to navigate common challenges and manage their care. Get more information about the PASSAGES Transition Program.
  • Other assistance: Whether you’re looking for additional information about your child’s diagnosis or emotional support, we are here to help. We also assist families in accessing school-based services to help children keep up with their studies. Explore the HOPE Behavioral Health, Neuropsychology and Education Service.

Aplastic Anemia and MDS Research and Innovation

CHLA is a leading member of the Pediatric Transplantation & Cellular Therapy Consortium (PTCTC), funded by the National Institutes of Health. This international research network is the largest of its kind focusing on blood and bone marrow transplants in children. We are exploring ways to make transplants safer and increase the likelihood of success.

Find out more about our research and clinical trials.

Contact us

The Cancer and Blood Disease Institute welcomes new patients, referrals and second opinions. Please call us to make an appointment.

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