A recent article in the Taunton Gazette describes the dilemma faced by the parents of four-year-old Jaxtien Miller who is a metachromatic leukodystrophy patient waiting for a stem cell transplant (SCT) that could slow its progression. Receiving the SCT procedure is a race against time as it cannot restore abilities that Jax already lost.
About Metachromatic Leukodystrophy (MLD)
A genetic mutation is the underlying cause of MLD. It causes a breakdown of myelin, the protective cover surrounding nerve fibers and nerve cells. The patient’s fibers and nerve cells are damaged as a result.
As the disease takes hold, within a year Jax may be unable to walk or even hold his head up. The life span of a child with this disease may be six to eight years. There is no cure.
Jax’s dad, Damien Miller, works for an automotive business that provides insurance for him but he has been unable to afford additional health insurance for his family. As a result, Jax is insured through MeridianHealth, an Illinois insurance company that is managed by Medicaid. MeridianHealth had sent Jax’s parents five denial letters before finally agreeing to cover his treatment at an out-of-network hospital in Pittsburgh.
The company’s denial letters stated that although it would not cover an out of state hospital it would cover the procedure at the Lurie Children’s Hospital in Chicago. There are only three hospitals in the country that specialize in Jax’s disease.
Jax’s family has been guided by Dean Suhr, President of the MLD Foundation. Suhr lost one of his three daughters at age ten through MLD soon after she received a bone marrow transplant. His second daughter, now 39, is also affected by MLD. She is nonverbal and is confined to a wheelchair.
The Foundation has advised Jax’s parents that although the Lurie hospital is one of the best children’s hospitals in the country, it has only performed two similar transplants. The Pittsburgh hospital, however, has seen one hundred and seventy-seven people with metachromatic leukodystrophy and has performed SCT on twelve patients.
The SCT recommended for JAX involves umbilical cord blood. Prior to the transplant patients will receive chemotherapy that clears out mutated bone marrow cells.
Chemotherapy with less toxicity is now being used by the Pittsburgh hospital for MLD patients. The results have been a survival rate of ninety-five percent.
About the Approval
Finally, after two months and five appeals, MeridianHealth approved the SCT at the Pittsburgh hospital. The approval coincided with both an article about Jax and the involvement of a state lawmaker, plus a nonprofit foundation. The family immediately flew to Pittsburgh where doctors will resume tests to determine whether he is still a candidate for the transplant. Jax had previously been disqualified by investigators of two clinical trials.
However, the final and difficult decision about whether to proceed falls on Jax’s parents. The SCT carries its own risk of disability or even death. Jax will spend up to six months in recovery at the hospital. There is no guarantee that it will slow the disease progress or whether it can add years to his life.
The cost of umbilical cord blood transplants plus the extended post-transplant hospital care can be anywhere from $100,000 to over $300,000.
Jax’s mother, Mercedes Boden, set up a GoFundMe site and the family also received support from the Cure MLD Foundation through Maria Kefalas, who co-founded the organization.
Kafalas’ nine-year-old daughter Calliope is an MLD patient but is not eligible for the transplant. She is non-verbal, fed through a tube, and requires ninety-six hours of nursing care each week.
Both Suhr and Kafalas agree since it is estimated that more than twenty-five million people in the U.S. have a rare disease, the difficulty of receiving out-of-state coverage is a major concern.
Staying within the network is safe when a patient requires a knee replacement or other common procedures. However, in the case of very rare diseases, there may be only two or three hospitals in the United States that have experience in treating a specific disease.
In order to encourage better patient care, states pay a flat monthly fee per member to managed care organizations such as MeridianHealth.
Those opposed to this system of payment point to missing and late payments from insurers to hospitals. They have also voiced concern about putting federal and state dollars into the hands of for-profit organizations. They believe that the capped payments may discourage carriers from covering expensive services.
The Health and Human Services (HHS) department is reviewing decisions denying services or payments that were eventually overturned through appeal. The department also believes that the current payment models in managed care are possibly an incentive to deny services to increase their profit margin.
One bright spot though is a new law regarding children with significantly complicated diseases who are Medicaid recipients. The law is slated to take effect in 2022. It will be up to each state to decide to join.