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A Father's Words Of Wisdom For Parents Of A Child With Hemophilia

Becoming a parent changes everything in life. This was certainly true for my husband, Jared, who'd always dreamed of being a father, despite having severe hemophilia B and frequent seizures. When our daughter, Cittie, was born, that dream came to life, giving him more purpose and even improving his health.

Reflecting on his journey, Jared often wonders what it would be like to raise a child with hemophilia. He certainly understands the challenges it entails.

In this column, Jared shares below some of his thoughts for parents of children with hemophilia. His advice is rooted in practical wisdom, aimed at helping them prepare for the realities ahead while keeping their expectations grounded.

Accept the journey, but plan ahead with flexibility

Parenthood is full of surprises. When you plan to have a child, you must accept the possibility of any situation, including that your child might have a health condition. This doesn't mean you need to have everything figured out ahead of time, but you should at least have a plan in place that you can adapt when necessary.

You also don't have to be perfect or have all the answers right away. Parenthood isn't about getting everything right the first time; it's about doing your best and being flexible when life throws you curveballs. When raising a child with hemophilia, sometimes things won't go as expected, and that's OK. What's important is to be prepared to adjust and adapt.

Let your child be more than hemophilia

One of the biggest lessons I've learned is that a person is so much more than their health condition. It's easy to let hemophilia take center stage in the parenting journey because it requires so much attention. But remember, your child is a person first — with their own unique interests, passions, and dreams. Don't let hemophilia define their life or overshadow the individual they are becoming.

Teach them to live, not just survive. Help them learn the life skills they'll need to adapt and thrive, even with hemophilia. It's not about shielding them from every possible risk, but teaching them how to manage their condition independently.

Focus on quality of life and emotional health

Raising a child with hemophilia isn't just about managing their physical health; it's also about nurturing their emotional well-being. The toll that a chronic health condition can take on a child's mental health shouldn't be underestimated. Physical limitations and medical treatments can sometimes make life feel overwhelming for them. As parents, it's crucial to ensure your child doesn't believe that life isn't worth living because of their condition.

Your role isn't just to help them manage hemophilia but also to support them in building a life full of joy, hope, and purpose. By addressing their emotional needs and ensuring that they know they are loved and supported, you'll help them develop resilience in the face of their challenges.

Prepare them for the future

One of the hardest but most important lessons as a parent is that you won't always be there for your child. This is true for all parents, but especially so for parents of children with hemophilia. Teach your child how to manage their condition so that they can live independently. They need to know how to handle life without you because one day, they'll have to.

At the end of the day, parenthood is messy. None of us have it all figured out. But you only get one shot at raising your child, so make the most of it. Learn from your mistakes, grow with your child, and give them the tools they need to lead a full and vibrant life.

Jared's message is simple: While the parenting journey may be difficult, it's also filled with opportunity. As parents, you have the unique chance to guide your child toward a future where they live not just in spite of hemophilia, but alongside it. Parenthood isn't perfect, but the lessons you learn — and teach your child — are priceless.

Note: Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to hemophilia.

Mother Of Ballerina Michaela DePrince Dies 1 Day After Daughter's Death At 29

The mother of the late ballerina Michaela DePrince died one day after the death of her adopted daughter. 

Elaine DePrince died Sept. 11 at 77 "during a routine procedure" that took place within 24 hours of her daughter Michaela's death, according to a statement posted to Facebook by Jessica Volinski, a spokesperson for the DePrince family. Michaela was 29.

Elaine had been battling "a hereditary disease and heart failure" for many years, and, per Volinski's statement, did not know that Michaela had passed away when she went in for her procedure.

Elaine DePrince died Sept. 11 at 77. Elaine DePrince / Facebook

"There has been a great deal of speculation online so the family would like to officially set the record straight about a few things. The last few days have been even more difficult than most people realize because the family has also been dealing with the death of Michaela's adoptive mother Elaine DePrince," Volinski wrote in the post.

"[Michaela's] mother Elaine died during a routine procedure in preparation for a surgery on September 11th. Michaela died before Elaine and Elaine did not know of Michaela's passing at the time of her procedure.

"As unbelievable as it may seem, the two deaths were completely unrelated," the statement went on. "The only way we can make sense of the senseless is that Elaine, who had already lost three children many years ago, was by the grace of God spared the pain of experiencing the loss of a fourth child."

Volinski 's post continued, "What the family is going through right now is truly unimaginably painful. Grieving two family members who died within a 24 hour period is tragic and devastating. We continue to ask for privacy and appreciate you directing anyone sharing incorrect information and speculation to this post."

Michaela DePrince performs with the English National Ballet in 2017 in London. Ian Gavan/Getty Images Elaine died one day after the late ballerina. Elaine DePrince / Facebook

A separate post on Elaine's Facebook page announced the news of her death.

"After many years of battling a hereditary disease and heart failure, we are saddened to bring you this news. Elaine DePrince passed away on September 11th at the age of 77," the post read.

"She is survived by her children Mia, Beelee, Jaye, Mariel, Amie and her two sons, Erik and Adam. The family thanks you for their privacy as they navigate this very difficult time."

A post on Elaine's Facebook page revealed she had been battling "a hereditary disease and heart failure" for "many years." Elaine DePrince / Facebook Michaela's final Instagram post on Aug. 9. Instagram / @michaeladeprince

On her Facebook page, Elaine described herself as "A former teacher, an author, a lyricist, and mother of 11." She also noted that she wrote a book, "Cry Bloody Murder," which she said was about hemophilia and HIV, and co-authored another book, "Taking Flight."

The Post has reached out to Volinski for further comment.

Michaela grew up as an orphan in Sierra Leone before being adopted by Elaine and brought to her home in Cherry Hill, New Jersey, joining Elaine's 11 other children (nine of whom were adopted). Elaine enrolled Michaela in a dance class soon after the adoption. 

"There was so much love right away," Michaela told Glamour in 2015. "I had never been surrounded by something like that."

Elaine and Michaela in 2017 in New York City. Jason Kempin/Getty Images

Michaela was internationally renowned for her work, having been a ballerina at the Dutch National Ballet and, as of 2021, a principal soloist at the Boston Ballet. She starred in the 2011 documentary "First Position" and was featured in the 2021 movie "Coppelia." She also performed on "Dancing With the Stars" during AT&T's 2012 spotlight performance and in Beyoncé's "Lemonade" film.

Beyoncé remembered DePrince in a post on her website. "Rest in peace Michaela Mabinty DePrince. Your courageous journey and beautiful dance as one of the world's best ballerinas, will stay with us always," Beyoncé wrote.

Michaela at the American Ballet Theatre Gala in 2022 in New York City. Getty Images for American Ballet Theatre Michaela was an internationally renowned ballerina. Instagram / @michaeladeprince

Michaela's death was confirmed in a post on her Instagram Friday. 

"With pain in our hearts, we share the loss of star ballerina Michaela Mabinty DePrince, whose artistry touched countless hearts and whose spirit inspired many, leaving an indelible mark on the world of ballet, and beyond," a statement posted by "Team MDP" said. 

"[Michaela's] life was one defined by grace, purpose, and strength. Her unwavering commitment to her art, her humanitarian efforts, and her courage in overcoming unimaginable challenges will forever inspire us. She stood as a beacon of hope for many, showing that no matter the obstacles, beauty and greatness can rise from the darkest of places."

News of Michaela's death was confirmed in an Instagram post on her account Friday. Instagram / @michaeladeprince

"Though her time with us was far too brief, her brilliance and legacy will continue to shine in the hearts of all who were touched by her story, for generations to come," the statement concluded. "Love and prayers go to her chosen family, friends, and those who loved her."

Michaela's cause of death is not known.

Gene Therapy For Hemophilia B Significantly Outperforms Factor IX Prophylaxis

Gene therapy for hemophilia B allowed almost three-fourths of patients to discontinue prophylactic factor IX therapy with no increase in bleeding, results of the pivotal BENEGENE-2 trial showed.

The annualized rate of bleeding for total bleeding episodes decreased by 71% after treatment with fidanacogene elaparvovec (Beqvez), while the mean annualized rate of bleeding for treated bleeding episodes was reduced by 78%, reported Adam Cuker, MD, of the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, and co-authors in the New England Journal of Medicine.

The results met prespecified criteria for noninferiority compared with factor IX prophylaxis and provided the basis for FDA approval of the therapy earlier this year.

"In this study, factor IX levels after fidanacogene elaparvovec therapy, as compared with prophylaxis, were associated with an amelioration of the bleeding phenotype and a significant reduction in the annualized bleeding rate and the annualized total factor IX consumption," the authors wrote. "These findings offer additional evidence that transduction of the FIX-R338L variant can produce hemostatic competence at the reported factor IX activity level."

"The majority (>80%) of the participants had factor IX activity in the mild-hemophilia range for a sustained period of 15 to 24 months, a finding that shows durable efficacy similar to that observed in other trials of gene therapy for hemophilia B," they added.

Standard of care for hemophilia B is episodic intravenous administration of factor IX replacement, Cuker and co-authors noted in their introduction. Current prophylactic therapies do not provide a cure, eliminate symptoms, or prevent joint damage.

Other non-factor IX agents that reduce antithrombin synthesis and monoclonal antibodies that neutralize the inhibitory activity of tissue factor pathway inhibitors have either been approved or are in advanced stages of clinical development, the authors noted. However, those agents still require regular administration.

Gene therapy can eliminate the need for ongoing treatment and the burden of regular disease management. The FDA approved etranacogene dezaparvovec (Hemgenix) as the first gene therapy for hemophilia B in 2022. Administered as a single infusion, the therapy consists of an adeno-associated virus (AAV) serotype 5 vector expressing FIX-R338L.

Fidanacogene elaparvovec delivers transgene production of FIX-R338L. In a phase I-IIa trial with long-term follow-up, the gene therapy led to sustained factor IX activity in the range of mild hemophilia to normal, associated with few episodes of bleeding and reduced factor IX prophylaxis. Results of the 15-patient study provided the basis for the phase III BENEGENE-2 trial that led to the therapy's approval.

BENEGENE-2 included men ages 18-65 with hemophilia B, defined as a factor IX level ≤2%, with at least a 6-month history of factor IX prophylaxis. After screening and exclusions (primarily for anti-AAV antibodies), 45 patients received a single infusion of fidanacogene elaparvovec, and 44 completed at least 15 months of follow-up.

The primary endpoint was the annualized rate of treated and untreated bleeding episodes from 12 weeks to 15 months after treatment, as compared with the factor IX prophylaxis lead-in period.

The results showed that the annualized bleeding rate decreased from 4.42 episodes at baseline to 1.28 at 15 months, a difference that not only met noninferiority but also superiority versus factor IX prophylaxis (P=0.008). At 15 months, the mean factor IX activity was 26.9%.

A total of 28 patients received glucocorticoid treatment for increased liver enzymes or decreased factor IX levels between 11 and 123 days. No infusion-related serious adverse events, thrombotic events, or malignancies occurred, as well as no development of factor IX inhibitors.

"The findings from this phase III study showed that fidanacogene elaparvovec had a favorable benefit-risk profile providing efficacy at one of the lowest doses of AAV-based gene therapy studied for hemophilia B," the authors concluded. "An ongoing extended follow-up study to 15 years after gene therapy will provide further insights regarding the effects of fidanacogene elaparvovec."

Charles Bankhead is senior editor for oncology and also covers urology, dermatology, and ophthalmology. He joined MedPage Today in 2007. Follow

Disclosures

The study was supported by Pfizer.

Cuker disclosed relationships with MingSight Pharmaceuticals, the New York Blood Center, Pfizer, Sanofi, and Synergy Research, as well as a patent/royalty/intellectual property interest.

Primary Source

New England Journal of Medicine

Source Reference: Cuker A, et al "Gene therapy with fidanacogene elaparvovec in adults with hemophilia B" N Engl J Med 2024; DOI: 10.1056/NEJMoa2302982.

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