A new twist on an old procedure cures kids with Sickle Cell Disease

Ten-year-old Paulette Amaechi and her mother, Felicia. Paulette was born with Sickle Cell Disease, but was recently cured with a bone marrow transplant. (Amaechi family photo)

WASHINGTON — Ten-year-old Paulette Amaechi stands with the poise of a tiny dancer. She has a shy grin, bright eyes and says with conviction, “I either want to be a doctor or a firefighter.”

The reason? She wants to save lives, much like the bone marrow transplant team that saved hers.

Paulette was born with Sickle Cell Disease (SCD) — a hereditary blood disorder in which red blood cells become misshapen and lose the ability to move freely through the body, clogging bones and slowly damaging vital organs. More than 100,000 people in the United States have it, and one in eight African-Americans is a carrier.

“It is a very bad disease,” says Dr. Allistair Abraham, an SCD specialist with the Children’s National Medical System. “It is chronic in that it slowly accumulates over time, and in the end it leads, unfortunately, to a poor quality of life and then early death.”

Abraham estimates that on any given day, Children’s National is helping 1,200 to 1,600 local kids with SCD. These patients are dealing with constant pain and disrupted lives.

Paulette’s mother, Felicia Amaechi, remembers the tears of joy when her daughter was born — and the sobs of worry that followed the SCD diagnosis.

“I was crying because I did not know what would become of her,” she says.

Felicia could not bear the thought that Paulette’s suffering would get worse, and so she began looking for answers. Ultimately, her quest led the Baltimore family to Children’s National, where Paulette got a bone marrow transplant when she was only 6 years old.

She became the kind of medical statistic parents dream of: Paulette Amaechi was completely cured, and now her story and her medical records are being used by researchers who are looking for the best ways to treat other kids with SCD.

Paulette’s case was one of 20 studied by researchers at Children’s and other medical centers in Atlanta and New York. All involved patients who received their transplants early in the progression of the disease, before there was any sign of organ damage.

The researchers wanted confirmation that bone marrow transplants – once used as a last resort for the sickest kids – could be a viable option as a pre-emptive treatment. They were struck by the results. Not one of the patients who underwent the procedure had a recurrence. Like Paulette, all were cured.

“We can transplant these patients and we can improve survival,” says Jacqueline Dioguardi, a physician’s assistant with the transplant program at Children’s National who worked on the study. “We can cure them before they have manifestations of their own Sickle Cell Disease, hopefully improving their quality of life overall.”

Currently, the average lifespan for a child with SCD is roughly 30 to 40 years. Dioguardi says with bone marrow transplants, these kids may be able to live into their 80s.

However, as good as the medical procedure may be, there is an obstacle with bone marrow transplants: finding an acceptable donor.

A close relative is usually the best match, but since the disease is genetic, and those who carry the trait cannot donate, the pool of likely donors for most kids is very small.

In this respect, Paulette Amaechi was lucky. Of her four siblings, one proved to be a perfect match: her college-aged sister Pearl.

Some of Pearl Amaechi’s bone marrow was extracted during an outpatient procedure and cells were transfused into Paulette, who had already spent months in the hospital receiving chemo that wiped out her immune system in preparation for the transplant.

More months of isolation followed while her immune system rebuilt itself.   Gradually, her life — and the lives of those around her — evolved to a new, pain-free normal.

“Today, Paulette is a new blessing to her family and we are grateful to God and her doctors,” says her father, Daniel Amaechi.

While the transplant team inspires Paulette, she is especially grateful for big sister, Pearl. One is small and one is grown, but they share a special bond.

“She’s my sister and I wanted to help save her life and keep her healthy,” Pearl says.

Paulette looks up at the now-grown woman who towers over her and whispers,  “She is so great — she just is.”

Follow @WTOP on Twitter and WTOP on Facebook.

© 2015 WTOP. All Rights Reserved.

Advertiser Content