Nigeria struggles with essential care for bleeding disorders as global medicine moves to gene therapy

Inside the living room of a home in Nigeria’s northern city of Kaduna, a newlywed couple looked at their third-born son and named him Onwuzueike, “death must cease.”

It was less a nomenclature and more a desperate petition.

They had already buried two sons before their tenth birthdays, one lost to a bike accident and another to what could have been a routine eye surgery.

Both were victims of an unceasing bleed that family rumours had instead attributed to “spiritual curse.”

But for the third son, Victor Ugochukwu, the curse would later find a name: Haemophilia A.

He was born into a body with a “Factor 8” protein level of 0.01 per cent, a misfortune that meant a fall at age five wouldn’t just result in a bruise, but a year-long confinement to bed, followed by two horrific encounters with an unorthodox bone-setter who would crack apart his blood-stiffened knees without anaesthetic.

“I might as well have died that day,” said Mr Ugochukwu, now 31, sighing and shuddering at the memory of being subdued by hefty men, the swollen legs bent so far backwards that his heels touched his thighs.

What the bone-setter did not know was that the knee joint was not stiffened from structural damage but was internally drowning in blood, a pool no amount of traditional manipulation could drain.

When bodily trauma causes tissue rupture, a natural clotting mechanism quickly plugs the wound to stop bleeding. This “coagulation” is an autonomic response for most people, but not patients with haemophilia, an inherited disorder in which wounds don’t clot properly, leading to prolonged bleeding.

The prevalence of haemophilia is estimated at one in 10,000 live births, affecting up to 465,000 individuals globally, according to data from the World Federation of Haemophilia.

Haemophilia A, which occurs when a clotting protein called “Factor 8” is dangerously low in the blood, is the most common form of the disease. It impacts about one in 5,000 to 10,000 male births.

The condition affects between 11,000 and 20,000 Nigerians, with over 90 per cent still undiagnosed and some 946 patients already identified, according to state sources and data from independent non-profit research.

Turning to traditional medicine

In Nigeria’s south-eastern state of Enugu lives Josephine, 38, a mother of three boys with Haemophilia A.

The first time she heard of the disorder was after her first son, Chidebere, was cut, from a doctor who himself doubted that an African could really present with what he had deemed to be “white people’s sickness.”

Eight days after a local midwife helped bring the boy to life, the circumcision bleed would not stop.

“The child was in diapers. The whole diaper was soaked with blood. He was pale. His eyes were wide, darting left and right. We knew something was wrong,” Ms Josephine narrated.

Transfusing the baby with “factor-rich” blood helped clot the bleed, but this was just the beginning.

In the sixth week, Ms Josephine would accidentally nick the baby’s flesh while trying to trim his fingernails, and the tiny cut would again not stop bleeding.

Panic set in when the toddler got knocked down a flight of stairs in pre-school, falling on his knee.

A warm massage only doubled the swell by morning. And while a doctor confirmed the knee wasn’t broken, he lacked the words to explain why it was “ballooning.”

Desperate for answers the clinic couldn’t provide, Ms Josephine turned to a local bone-setter whose attempt to stretch out the knee only “worsened” the internal bleeding.

It took the bone-setter’s own bafflement and a fortuitous referral to a teaching hospital to finally replace traditional ointments with the science of haematology.

When Chidiebere was injected with a vial of donated Factor VIII protein by the non-profit Haemophilia Foundation of Nigeria, the agony that had defied every herb and bandage vanished in mere minutes.

Haemophilia is a sex-linked disorder carried on the “X” chromosome. Because males have only one X chromosome (XY), inheriting a single defective gene from their mother causes the disease.

Women have two X chromosomes (XX). A healthy gene on one compensates for a defective one on the other, only making the woman a carrier. She may show no symptoms, but still has a 50 per cent chance of passing the hemophilic gene to her sons and a 50 per cent chance of also making her daughters carriers.

Per medical research, about one-third of babies diagnosed with haemophilia have no pre-existing family history of the disorder, signalling spontaneous mutations as culprits.

The World Federation of Haemophilia (WFH) database shows Nigeria had 758 Haemophilia A patients as of 2024, 33 per cent of whom were between the ages of five and 13. Some 30 per cent are adults, aged 19 to 44.

Ms Josephine’s own parents had three male children, none of whom were hemophilic. Her sister had four boys, none of whom were also hemophilic.

Medication priced beyond reach

The treatment options for haemophilia in Nigeria are primarily clotting factor concentrates, which are administered either on-demand during crisis or at intervals as low-dose “prophylaxis” to prevent bleeds.

There are also newer but expensive non-factor therapies like the monoclonal antibody Emicizumab, whose longer-acting effect, clinicians say, could give haemophilia patients a totally normal quality of life.

But access is severely constrained by a lack of government subsidisation, forcing patients to rely perennially on donations from international non-profits due to the prohibitive cost of commercial purchase.

Market research says a 30mg vial of Emicizumab costs up to $3,500, about N5.25 million at N1500/USD. Some patients, haematologists confirm, require up to seven vials a month, depending on body mass.

A 500 IU vial of the “Factor 8” concentrate could cost up to $1,500 at commercial price. A standard dose to stop a medium-severity bleed for an average adult in on-demand treatment can be up to 1,500 IUs.

Austin Usifo, the head of marketing at Biofem Group, said the pharmaceutical company does not import haemophilia medication because the product does not enjoy substantial commercial demand.

Unsubsidised commercial access is so prohibitively expensive that families are confronted by financial ruin, waiting on donations, or a patient’s severe disability or death.

Despite the universal coverage mandate of Nigeria’s National Health Insurance Authority (NHIA) Act, the legislation excludes high-cost treatments for rare conditions such as haemophilia.

Factor VIII and IX coagulants are listed among essential medicines to which health institutions must ensure access, but they are excluded from the NHIA drug list, meaning insurance does not cover them.

In 2022, Nigeria captured bleeding disorders in its national non-communicable diseases strategic plan. But an implementation guideline is required before state funds can be set aside for factor procurement.

Living dangerously on donations

Sanusi Omobolaji, 32, had just returned to his home in Ogun from a long-dreaded trip to Akure. In under 24 hours, the adrenaline of travel had given way to the throbbing heat of a spontaneous ankle bleed.

Mr Omobolaji was stranded; the empty Factor 8 vial on the creative designer’s work station had been his last. And the nearest replenishment was only in a four-hour traffic-fraught trip to Lagos that he could no longer manage.

He lives in a clinical blind spot. In the last 12 months, he has not attended his quarterly haemophilia clinic at the Lagos University Teaching Hospital, where he could have received a take-home packet.

He moves with a cane, favouring a right knee that has bowed under the weight of decades of internal haemorrhaging, admittedly unable to walk for up to 10 minutes without his body demanding a rest.

“All my joints, both major and minor, are affected by deformity,” he noted with a sobering honesty he rarely affords himself. “Whether sleeping, lying down, walking, or sitting, I’m constantly in pain.”

For Mr Omobolaji, care is not merely about the existence of medicine, but also the ability to survive the terrain required to reach it. He was considering the difficult interstate trip to LUTH. Unknown to him, at that time, even the hospital’s model haemophilia treatment centre at Idi-Araba, Lagos, was out of stock.

LUTH is one of the 17 hospitals designated by the Haemophilia Foundation of Nigeria as a Haemophilia Treatment Centre (HTC).

LUTH’s haemophilia clinic, as of January, reported 136 registered patients, with about 70 in active care, meaning they must undertake two to three clinical contacts in any year for checkup and education.

HTCs receive a quarterly shipment of WFH products through the HFN, alongside training, to provide comprehensive care for patients, ideally “supposed” to include orthopaedic, physiotherapy, mental health counselling, and social support.

But donations from WFH’s humanitarian aid programme have been the sole source of life-sustaining factor concentrates available to these patients and all others among the 946 registered with Nigeria’s 17 HTCs, an anomaly even the global non-profit itself admitted it has been fighting to correct and discourage.

A donation cycle begins with securing a NAFDAC nod to import, followed by a standoff with Customs at an airport cargo terminal.
Once liberated from the tarmac, the temperature-sensitive vials are hauled inland on a cold truck to the Haemophilia Foundation of

Nigeria’s headquarters in Kaduna, from where they are distributed to HTCs.

And not even the coveted donor products are spared misfortune.

Benjamin Clousier, communications manager at the WFH, said the shipment approval timeframe with Nigeria’s health authorities has significantly increased to three months, stalled by demands for documentation “unrelated” to core clearance priorities like the medicine’s safety and efficacy.

This drag, he noted, has forced the UN-affiliated non-profit to slash its shipping frequency from quarterly to twice a year.

“Several patients are on prophylaxis. If, for any reason, the donations were delayed, these Nigerians will have to stop treatment, which will expose them to life-threatening bleeds,” he stated in an email.

The resultant defaults in quarterly shipments, the director of LUTH’s HTC, Titi Adeyemo, said, force the treatment centre to reserve prophylactic care for only children under age 12.

It means the adult patients do not have access to preventive prophylaxis.

“They’re treating bleeds when the standard of care should be prevention,” the doctor said.

“More bleeds mean more joint disease. Eventually, the joints fail, and you start using a walking aid or wheelchair.”

Diagnostic capacity is still a luxury

For Iyabo Akinleye, the true cost of Nigeria’s healthcare deficit can be measured in the several years of clinical guesswork she endured.

As a kid, she bled a lot, enough so that her parents panicked. After immunisation shots in infancy, she bled. When she was teething at six months old, she bled. During tooth-shedding at 7, she also bled.

Every growth milestone meant a frantic race to the hospital ward.

When a seven-year-old Iyabo was put in bed at her family’s Kaduna home after a chloroquine injection for malaria, her mother expected the restorative sleep of a recovering child.

Instead, she woke to find her daughter in a pool of red. What she had mistaken for a child wetting the bed turned out to be blood slowly draining out from a needle site that a simple tuft of cotton wool should normally have sealed.

Iyabo was thereafter subjected to a strict list of Dos and Don’ts, as her worried parents sought answers.

Puberty would usher in unusually heavy, long-lasting menstrual periods that had her skipping school.

In search of a diagnosis, she was at the Obafemi Awolowo University Teaching Hospital in Osun, the University of Ilorin Teaching Hospital in Kwara, and the Ahmadu Bello University Teaching Hospital in Kaduna.

“I told them I had a bleeding problem at the time. They didn’t know what was wrong,” Iyabo recounted.

Nighttime offered no reprieve, as Iyabo would wake several times to expel blood that had pooled in her throat while she slept. A doctor had hinted at haemophilia. But it was with repeated therapeutic trials that her condition would be pronounced with fairly reasonable certainty as Von Willebrand’s Disease (VWD).

Research says VWD is a more common bleeding disorder, affecting one in 100 people. In Nigeria, however, there were only 18 confirmed cases as of 2025, a reality experts attribute to a lack of diagnostic capacity.

VWD patients either lack or have low levels of a clotting protein called von Willebrand factor, or it doesn’t work properly. For a person to make a clot, VWF binds to factor 8, another clotting protein, and platelets in blood vessel walls. In a normal person, this process forms a platelet plug, which helps a bleed stop.

Iyabo was subjected to a panel of basic blood tests that established she had 7 IU/dL of the VWF, where normal levels should be between 50 and 200. None of the hospitals she visited could perform the gene tests needed to determine why her VWF levels were low and identify her type of VWD.

Titi Adeyemo, LUTH’s HTC director, said Nigeria’s tertiary hospitals and labs lack the infrastructure to reliably test for bleeding disorders, with even privately-run clinics resorting to sending samples abroad.

“Hence, we rely on therapeutic trials. There are different bleeding disorders and different treatment options. If we give them one treatment and it works, we can focus on it. This is how we’ve been going.”

A coagulometer is a machine used to measure blood clotting and diagnose bleeding disorders such as haemophilia. But it was only in 2025 that LUTH acquired one through a donation from the Danish non-profit Novo Nordisk, allowing it to conduct factor assessment and name a condition, but not much else.

Nigeria’s maternal mortality is high, with UN data showing that one in 100 women dies in labour or in the following days, and postpartum haemorrhage is identified as one of the culprits.

Iyabo, now 39 with two dissuasive childbirth experiences that had her family on edge, thinks bleeding disorders could be a factor.
She fears that Nigeria may never know for sure without data and the proper diagnostic infrastructure.

Nigeria sidelines gene therapy as price tag stalls access

Haemophilia is due to a missing or defective gene that prevents the body from making a clotting protein.
Beyond “miracle” injections like Emicizumab that rebalance the system, WFH data show that gene therapy offers a longer-lasting treatment, enabling the body to naturally manufacture the missing clotting factor.

As the delivery medium or “vector”, scientists use ”harmless“ Adeno-associated viruses, infused with a working copy of the missing clotting gene.

The modified virus is delivered to a patient via a single intravenous injection, then travels through the blood to the liver cells, which are the body’s natural factories for clotting factors. The gene, once added, remains in the cells while the vector leaves the body through urine, faeces, blood, saliva, and semen.

Factor levels, successful clinical trials show, begin to rise after three to four weeks of gene therapy.

Australia’s Mark Lee is one of the most famous success stories for haemophilia treatment with gene therapy. After receiving the injection in a trial, his clotting factor levels were reported to have moved into the normal range. He went from needing factor infusions thrice a week to living without any bleeds.

In the US, three landmark gene therapies have been approved for haemophilia care, including Hemgenix and Beqvez for Haemophilia B, along with Roctavian for Haemophilia A.

The therapies, priced between $2.9 million and $3.5 million per dose, are transforming haemophilia care.

In the UK, trials at the University College London and St. Jude Children’s Research Hospital reported that haemophilia patients have maintained stable clotting levels for over 13 years after a single dose.

A study of Haemophilia A patients three years after Roctavian treatment reported that average Factor VIII levels were 42 per cent after one year, dropped to 23 per cent in two years, and dipped further to 15 per cent in year three.

Due to variations in outcomes, clinicians say the medical community is focused on long-term monitoring to see whether the clotting factor levels produced by these “one-time” shots will stabilise over the decades.

Interestingly, artificial intelligence is emerging for personalised treatment and usage in conjunction with point-of-care ultrasound to quickly detect early joint damage for inherited disorders like haemophilia.

But Megan Adediran, founder and executive director of the HFN, said prohibitive costs, infrastructure gaps, and competing health priorities hinder Nigeria’s deployment of these innovations.

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Emmanuel Kolawole, a researcher at the University of Connecticut, said demand for pricey gene therapy is low even in high-income markets, citing Pfizer’s discontinuation of global development of Beqvez.

Afees Olanrewaju, a medical scientist at Nigeria’s Babcock University, worries that big pharma is making limited efforts to facilitate licensing, regional manufacturing, and competitive pricing in the global south.

A geneticist, Christian Happi, in a lab on the red earth of Osun State, pioneered a landmark, albeit donation-funded, public health programme using CRISPR to surveil for deadly diseases.

Yet, Nigeria has not notably begun exploring this gene editing tool in actual treatment, beyond fielding patients for international clinical trials.

At LUTH, Titi Adeyemo watched these gaps consume a patient, Michael, who, after a lifetime of undiagnosed agony, presented with a leg so ravaged by gangrene that only amputation could save him.

Insisting he would rather “die whole,” Michael, 22, signed himself out into the silence of a certain death.

“I felt very hurt,” surgeon Titi Adeyemo admitted, wincing from behind her office desk. “You wonder if you could have done more.”
Access to even the most essential haemophilia care in Nigeria remains poor, devastating the education, sex life, and mental health of many, alongside the already strained finances of their families.