Givlaari Beyond the Trials: Real-world Patient Experiences

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by Claire Richmond |

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Givlaari (givosiran) is the most exciting development in the acute hepatic porphyria (AHP) community since Panhematin (hemin for injection) was approved under the U.S. Orphan Drug Act in 1984. I started treatment earlier this year, and I’m now five months in. When I reflect on my experience with acute hepatic porphyria (AHP) and Givlaari, there’s so much to consider.

First off, it’s not a cure. I’m not sure who started that rumor.

Let me be clear, I absolutely want to live in a world without AHP. But a laserlike focus on finding a cure is only helpful if you’re a researcher. As a patient, that outcomes-oriented mindset is challenging.

A world where no one is affected by AHP would be a utopia, where bodies aren’t hijacked by paralysis, and torturous pain doesn’t overpower someone’s life. Unfortunately, this future is both undefined and elusive. I don’t even know if my current flare will allow me to grab coffee with friends tomorrow morning.

For the sake of my delicate mental state, I can’t live in an AHP-free future that doesn’t yet exist. Because, while researchers are looking for a cure for AHP, I’m navigating the day-to-day challenges of chronic symptoms on top of frequent, unpredictable, acute attacks — sometimes holding on for dear life. A cure doesn’t help me in those moments.

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So, when I started hearing the word “cure” through the porphyria patient grapevine, I was both excited and wary. Some folks have done well on the treatment, and I’m psyched for them. But I’m five months in, and my life hasn’t dramatically changed.

Changes 5 months in

The biggest difference in my life is that I no longer receive weekly prophylactic heme, because Givlaari’s job is prevention. Without it, my attacks seem more dramatic, and hit suddenly. Because of this, my abdominal pain seems worse. For instance, in eight of the last 10 days, I’ve experienced abdominal distention, blinding pain, and the feeling of pressure, like an alien trying to push its way out of my stomach. I need rescue heme several times a month.

My symptoms have shifted, but I wouldn’t describe them as “improved.” For instance, instead of constant constipation, I have chronic nausea. I still have migraines and insomnia. My aminolevulinic acid and porphobilinogen (ALA/PBG) levels have come down a bit, but remain elevated.

With time, will Givlaari prevent my attacks? I will stay on it to find out, as long as I don’t experience adverse effects and my insurance continues its coverage of both the shot and crisis heme.

Concerns from others

The real-world effectiveness of Givlaari is coming to light, and it’s the hot topic among my patient friends. Based on conversations, it seems like ALA/PBG levels lowered in line with study results, but cannot be relied upon to indicate improvements. This is particularly problematic in attacks, if a provider is relying on elevated levels before administering rescue treatment.

A handful of friends on Givlaari struggle to be understood by providers during an attack, now that their levels are normal. A couple told me they are experiencing elevated homocysteine, and almost all have missed a dose due to trouble with health insurance certification.

A friend on the East Coast had a great initial experience on Givlaari, going from weekly heme infusions to one every other month. A year in, an insurance authorization issue delayed a dose. The following month, she experienced blood pressure spikes, abdominal pain, and what she refers to as “nausea from hell.” Last spring, after nearly a year and a half on the treatment, she was hospitalized for uncontrolled hypertension and pain.

A friend on the West Coast hoped Givlaari would help her gain stability, but still experienced dramatic attacks during her cycle. Now that her ALA/PBG levels are in the normal range, she has what she calls a “double fight” to get the several doses of heme she requires during a crisis. She is missing her dose this month due to problems recertifying her health insurance coverage.

Like me, both are choosing to continue their treatment for now. We are learning firsthand how short-term efficacy from a research study can translate to long-term effectiveness in a world that’s messy and uncontrolled. We will keep learning and recording our symptoms. We will report them if they get serious. At the same time, we will continue making lifestyle modifications that support attack prevention.

In addition to a cure, I hope more researchers will study the most disruptive and psychologically impactful aspect of our day-to-day lives with AHP: acute and chronic porphyria pain. In the meantime, I will continue recording my symptoms and reporting back.

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Note: Porphyria News 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 other 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 Porphyria News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to porphyria.

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