Children with pediatric bone diseases can experience a range of symptoms including severe bone pain, stunted growth, debilitating fatigue and multiple fractures. These symptoms greatly impact a child’s ability to attend school, be active and have fun with friends.
There are challenges in treating these children because conventional treatments can have many side effects or be ineffective. And some rare bone diseases didn’t have any treatments available until recently.
The Pediatric Metabolic Bone Clinic at Children’s Health℠ helps these children by providing cutting-edge treatments for common and complex metabolic bone diseases. The clinic is part of the nationally recognized Pediatric Endocrinology Program at Children’s Health.
“We treat some of the rarest bone diseases using the latest infusion and injection therapies,” says clinic director Muniza Mohammed Mogri, M.D., a Pediatric Endocrinologist at Children’s Health and Assistant Professor at UT Southwestern. “These treatments ease and sometimes stop symptoms, allowing a child to participate in sports and enjoy their childhood.”
Here, Dr. Mogri shares two innovative treatments that are making a difference in children’s lives, plus case studies that illustrate their impact.
1. The latest treatment for hypophosphatasia (HPP)
Hypophosphatasia is a rare inherited condition that can cause life-threatening respiratory failure soon after birth. It occurs when a genetic mutation affects the body’s ability to make an enzyme that’s essential for bone and teeth mineralization. As a result, children with HPP often have soft bones that break easily. Some have bowed or knocked knees. These children are often small in stature with short limbs and an abnormally shaped skull.
In October 2015, the Food and Drug Administration (FDA) approved asfotase alfa (Strensiq®), the first and only enzyme replacement therapy for HPP. “Before this drug became available, a child with HPP faced a lifetime of bone pain and fractures,” says Dr. Mogri. “Depression often develops during adolescence and young adulthood because of the negative impacts on quality of life.”
Case study: Asfotase alfa injections for HPP
The future is brighter for children with HPP thanks to this new therapy. Dr. Mogri notes the remarkable turnaround of one elementary school-age child who started the three times per week asfotase alfa injections last year. “This child had such extreme bone pain and fatigue that they slept nearly 18 hours a day,” says Dr. Mogri. “They couldn’t stay alert during speech therapy sessions intended to help with speech delays. Their skeletal development was that of a child one year younger.”
After starting asfotase alfa treatments, the child now has the energy to attend school and play with friends. Their speech and motor skills have drastically improved. “They’re able to function at their age level, both physically and socially,” says Dr. Mogri, who sees the patient every four months for checkups at the Metabolic Bone Clinic.
Dr. Mogri and a team of nurses at the clinic make sure parents are comfortable giving their children these injections until their child is old enough to administer the shot themselves. “Someone from our team is available 24/7 to address any issues or concerns,” she says.
2. Help for children with chronic recurrent multifocal osteomyelitis (CRMO)
CRMO (also called chronic nonbacterial osteomyelitis or CNO) is a rare autoinflammatory syndrome that develops during childhood, causing inflammatory lesions in the bones. These noninfectious bone lesions cause severe bone pain, swollen joints and fatigue. A child with CRMO may have slowed growth and joint contractures that affect mobility.
“Some children with CRMO are in too much pain to walk,” says Dr. Mogri. Conventional treatment includes nonsteroidal anti-inflammatory drugs (NSAIDs) given in combination with immunosuppressants, such as adalimumab (Humira®).
At the Metabolic Bone Clinic, Dr. Mogri treats pediatric patients who don’t respond to this first line of treatment or who experience treatment side effects. Patients receive outpatient infusions of zoledronic acid (Reclast®) every three to six months. This antiresorptive medication prevents the breakdown of bones and improves overall bone strength.
Case study: Zoledronic acid infusions for CRMO
Zoledronic acid has FDA approval for osteogenesis imperfecta in children, but not CRMO. However, the drug’s benefits often outweigh the risks for certain pediatric CRMO patients, says Dr. Mogri. She cites one example where a teenager had complete resolution of inflammatory bone lesions and bone pain after receiving just one full infusion (initially spread out in two smaller doses to ensure a child doesn’t have an adverse drug reaction). “This child no longer needs treatments,” she says.
In another case, a preteen was able to reduce their NSAID use from three times a day every day to one dose every few weeks. “NSAIDs ease pain and inflammation, but their overuse can cause gastrointestinal problems like ulcers,” says Dr. Mogri. “Zoledronic acid infusions reduce this risk of complications while easing or resolving a child’s CRMO symptoms.”
The bone clinic also uses zoledronic acid infusions as an off-label treatment for pediatric osteoporosis.
Why choose Children’s Health Metabolic Bone Clinic
The Pediatric Metabolic Bone Clinic takes a multidisciplinary approach to care. “When appropriate, we partner with pediatric rheumatologists, neurologists, neurosurgeons and orthopedists,” says Dr. Mogri.
Children and their families also benefit from a care team that includes physical therapists, nutritionists and social workers.
In addition, the bone clinic team provides advanced care, including zoledronic acid infusions, for children with Duchenne muscular dystrophy (DMD). Children’s Health is home to the only Parent Project Muscular Dystrophy (PPMD) certified Duchenne Care Center Program in Texas.
Children with DMD have an increased risk of low bone mineral density and vertebral fractures due to long-term use of steroids. Advanced nurse practitioners at the clinic ensure infusions take place on schedule and serve as a resource for families before, during and after the transfusions.
The Pediatric Metabolic Bone Clinic also provides advanced treatments for children who have:
- Achondroplasia
- Fibrous dysplasia
- Hypocalcemia and hypercalcemia
- Low alkaline phosphatase levels
- Low bone mineral density
- McCune-Albright syndrome
- Multiple (two or more) long bone fractures
- Osteogenesis imperfecta
- Osteopenia and osteoporosis
- Paget’s disease
- Rickets, including hypophosphatemic rickets
- Vertebral fractures
Learn more about our nationally recognized Pediatric Endocrinology Program at Children’s Health