Patient perspective: Thrombotic thrombocytopenic purpura (TTP)

INTRODUCTION — This topic was written by an individual patient diagnosed with thrombotic thrombocytopenic purpura (TTP). It is intended to offer clinicians insight into the experience of a single individual from that individual's point of view. This description of a particular patient's experience is not intended to be comprehensive or to provide recommendations regarding diagnosis, treatment, and/or medication information. It is not intended to be medical advice or to be a substitute for the medical advice, diagnosis, or treatment of a health care provider based on the health care provider's examination and assessment of a patient's specific and unique circumstances.

For related clinical topics, please see:

●(See "Diagnostic approach to suspected TTP, HUS, or other thrombotic microangiopathy (TMA)".)

●(See "Diagnosis of immune TTP".)

●(See "Immune TTP: Initial treatment".)

●(See "Immune TTP: Management following recovery from an acute episode and during remission".)

●(See "Immune TTP: Treatment of clinical relapse".)

●(See "Hereditary thrombotic thrombocytopenic purpura (hTTP)".)

UpToDate also offers patient education materials. "The Basics" topics are short overviews written in plain language, at the 5th to 6th grade reading level, that answer the main questions a patient is likely to have about their condition. You can share this content directly with your patients:

●(See "Patient education: Complete blood count (CBC) (The Basics)".)

●(See "Patient education: Oral steroid medicines (The Basics)".)

A PATIENT AND A PHYSICIAN — I was a TTP patient before I became a physician. My first episode occurred in 1990 when I was a college student, my second and third as a medical student. After the last episode, in 1996, I have not had a relapse.

I survived TTP three times because I was lucky enough to find a hematologist who had the knowledge and experience to treat me at lightning speed. The outcome could have been different.

I am now in my early 50s and a busy primary care physician. My experience with TTP impacts numerous small decisions I make and has made me a better doctor. (See 'Impact on life and work' below.)

THREE DISTINCT PRESENTATIONS OF THE SAME CONDITION — Each TTP episode presented differently. In the first, I did not have an accurate diagnosis; in the second, I was diagnosed in the nick of time; and in the third, I underwent active monitoring during a high-risk pregnancy.

Treatments were also different, with identification of plasma exchange as an effective therapy and my fortuitous connection with one of the world's TTP experts.

First episode (college student) — It started innocuously; I was tired. I had just arrived home from college on a semester break, and the fatigue didn't seem unusual.

My parents started to become concerned when my energy level was too low to do anything other than sleep for almost a week. By that time, I had an episode of gross hematuria and developed bruises on my legs that were coalescing and increasing in size. Even with no medical background, we knew something was wrong.

Our family pediatrician fit me into his busy schedule. I was a healthy 20-year-old with no medical history other than an occasional sports injury. He asked a few questions, listened to my heart and lungs, looked at the bruises on my legs, and ordered a blood count. He thought anemia was the most likely diagnosis. Then my labs came back showing low platelets and low hemoglobin. He called and told me to see an internist that afternoon.

The internist repeated the blood count and sent me to the hospital. I didn't want to go. I was a tired college student; I just wanted to lie on the couch. But I knew this was not normal, and it was frightening.

The hospital admission was a bit of a blur, with attempts to rule in or rule out various diagnoses. Specialists came and went. Diagnoses like leukemia were mentioned, and I had a bone marrow biopsy. Maybe it was immune thrombocytopenia (ITP). Steroids were started, and my platelet count started to improve. A hematologist thought I might have TTP, a much more concerning diagnosis with a high fatality rate. Another specialist said, "This can't be TTP, or you'd be dead."

This episode occurred around the time plasma exchange was recognized as a possible treatment for TTP, but plasma exchange then was not what it is today. The apheresis machine was huge, with knobs and lights; it took a half-hour to set it up and required a long consent form. It felt like an experimental therapy. This was also in the early years of the human immunodeficiency virus (HIV) epidemic, and exposure to multiple plasma donors was not to be taken lightly. I declined plasma exchange, to the chagrin of some of the specialists. But the internist supported my decision, and I recovered without it.

Since the steroids seemed to have helped, the presumptive diagnosis was ITP, a more common disorder with a much better prognosis. I was treated with steroids for an entire year, during which I had all the classic steroid side effects: mood swings, crying for no reason, brain fog, edginess, fatigue, central weight gain. I was given azathioprine to allow a lower steroid dose. This is not the preferred approach for ITP (or TTP) today, but at the time it was state of the art.

I knew that another episode (of whatever I had) could occur at any time. I was constantly going to the lab for blood count monitoring. I felt somewhat better, but I knew I was not myself due to the steroid side effects. It was hard for people to understand how sick I was because I looked fine on the outside. With the help of my family, I got through it.

Second episode (medical student with a one-year-old) — The second episode, a few years later, was both scarier and luckier. I was a medical student with a one-year-old child (following an uneventful pregnancy), on a family vacation in rural America. I again developed confluent bruises on my feet, this time followed by visual changes and trouble focusing.

What happened next probably explains why I am here to tell my story today. I knew I needed to be seen by someone who understood platelet disorders and how to treat them, but I was far from home. I was lucky to be a medical student and could ask the faculty for recommendations, and I was referred to one of the world's experts in platelet disorders who happened to work nearby.

I went to see the platelet expert the next day. I was so anemic I could barely walk from the parking lot to the emergency department (it turned out my hemoglobin was 6 g/dL). Initially he thought I had ITP (the working diagnosis from the previous episode), but as soon as he saw the severe anemia, thrombocytopenia, and schistocytes on my blood smear, his entire demeanor changed, and this became a life-or-death, "get this kid in the hospital" situation. I was getting plasma exchange within a few hours. Now that I know how hospitals function, I am even more impressed with the speed at which he got me diagnosed and treated.

Plasma exchange is not fun. You sit still for hours as your entire blood volume passes through the apheresis machine and is routed back to you, minus your own plasma, which has been replaced by liters of donor plasma. The machines back in the early 1990s made your entire body vibrate. You are cold, no matter how many heated blankets the nurses pile on top of you. Your lips tingle from hypocalcemia. As you sit there, you wonder, how many people donated this much plasma? What was the exposure risk for each one? What new diseases might have entered the blood supply that are not yet being screened?

At the same time, knowing that things could have turned out differently – how sick I was and still able to walk away – fills me with tremendous gratitude. It makes some of the frustrating aspects of my care, such as having a surgical resident put in a triple lumen central line that could not be used for plasma exchange, seem inconsequential. As I improved, the hematologist educated me about TTP, its treatment, and what to expect in the future. He gave me more than just good care; he gave me confidence that, in the right hands, TTP is treatable.

Third episode (third trimester of pregnancy) — My third episode was in 1996. This time we knew the diagnosis. It was clear I'd had TTP twice already. I was pregnant with my second child and seeing a high-risk obstetrician who monitored my blood counts frequently due to the risk for relapse during pregnancy.

Early in the second trimester, my platelets started to drop. Maybe it was just gestational thrombocytopenia, or maybe it was the beginning of another episode. I had no more fatigue than usual and no bruises. We watched and waited.

My platelet count continued to decline, and plasma exchange was proposed. But I very badly did not want to undergo plasma exchange while pregnant. Methods for screening the blood supply were not as sophisticated as they are now, and the risk of HIV from donated plasma in a large urban center was on my mind, more for my baby than for myself.

I started taking steroids at approximately 30 weeks, to treat possible TTP and to mature the baby's lungs if I needed to deliver early. Things held for a while, but anemia and thrombocytopenia worsened, and I developed subtle neurologic changes (strange visual effects and trouble focusing like I'd had before); it was clear we could not watch and wait any longer. I was induced at 35 weeks. Within two hours after delivery, I was hooked up to the apheresis machine, with the knowledge that my child was healthy and would not be exposed to donor plasma.

That was over 25 years ago. It was my last TTP episode. Why I had three episodes and no more is a mystery. Maybe it was because I avoided quinine. Maybe it was the intravenous immune globulin (IVIG) I received during my third episode. We don't know; risk factors and prevention of relapse in TTP are active areas of investigation.

IMPACT ON LIFE AND WORK — The more time without an occurrence, the less concerned I have become. I take no medication for TTP and do not routinely see a hematologist.

However, TTP is never too far from my mind. I know what it is like to almost die, and no matter how far into the past the episodes recede, the memory of that feeling persists.

●Costs and burdens:

•When I meet a new doctor, my antenna is up to determine whether they will take me seriously. If not, I move on.

•If I feel tired for more than a few days, I ask my physician to run a complete blood count (CBC). To some, this may seem like paranoia and unnecessary testing, but from my perspective, it is essential for peace of mind.

•Travel carries additional stress and the need to plan thoroughly. When deciding where to go on vacation, I only choose places where I know I will be near medical care (including plasma exchange if needed). Sometimes I get a pre-travel CBC. I carry a wallet card that explains my diagnosis and the need for urgent plasma exchange if I have a TTP episode.

•I avoid using certain electronics, since the strong vibrations remind me too much of the early apheresis machines that made my entire body vibrate.

•I do not drink tonic water (or anything else with quinine).

•There was a financial impact following my TTP admissions. Getting sick is expensive. Even before the modern era of co-insurance and out-of-pocket minimums, I was required to pay a substantial sum over the course of many years as a medical student and resident.

●Positives:

•Having TTP did not stop me from having a family and a career. Far from it. I am a busy full-time physician with two full-grown children.

•Being sick made me a better doctor. I pay attention to patients' questions and concerns, something I learned from my first internal medicine physician who stood up to specialists on my behalf. I learned that it is okay not to know the answer medically, but you'd better know when to phone a friend or seek out a higher level or care for your patients when warranted.

•I received excellent medical care from knowledgeable and compassionate physicians. Receiving such excellent care made me incredibly grateful. I would do anything for my hematologist, including writing this perspective to share my story.

LESSONS FOR THE CARE TEAM

●Rare diseases are challenging – Fatigue and bruising are incredibly common, and TTP is extremely rare. But it happens. Remember to consider TTP in the diagnosis of an acutely ill person, especially if another diagnosis is not obvious. Keep TTP in your differential diagnosis of sepsis, alcohol intoxication, subtle neurologic findings, and other common presentations.

Having a support group is very helpful for a rare disease, preferably a group that includes physicians knowledgeable about the disease. It's a reminder that you are not alone. And it helps to be able to look across the room (or on the screen) at the faces of everyone who has survived their episode(s).

●Primary care is essential – Specialists know the disease; primary care doctors know their patients.

Primary care doctors can be incredibly busy and may feel obliged to defer to specialists, but they also may be the only person who really knows the patient and is familiar with their personality and values. This is especially valuable in the midst of a crisis. During my first episode, my internal medicine doctor stood by me when I didn't want to do plasma exchange. I knew I had someone in my corner. The internist may not have had all the answers, but they were willing to support my decision to avoid a frightening and (at the time) high-risk procedure. This also gave me confidence to accept plasma exchange during subsequent episodes.

●Subtle symptoms can have not-so-subtle consequences – Brain fog is real. After an episode, reading and concentrating were challenging. I could read a sentence or a paragraph, close the book, and have no idea what I had read. I had to develop external memory systems and pre-plan my day: keys, textbook, notebook, written schedule; check the car for gas; get the groceries. Using external memory systems takes more energy than it should.

●Communication matters – As a patient, it can be hard to balance realistic expectations and self-advocacy when you are sick, scared, and lacking expertise in a condition. It sounds obvious, but what may be important to the patient is not always the same as what is important to the care team. Take time to answer those otherwise unconcerning questions. Learn about your patients as people.

It helps to hear the big picture, game plan, and expected outcomes. Convey the uncertainties with compassion. Remind patients that not everything they read on the internet is accurate, and that doctors speak in averages; they cannot always predict what will happen to an individual patient.

Treatment of TTP requires a lot of coordination. There are plenty of places for errors, missteps, and miscommunication. If you do not know the right answer, get it.

●Risk of recurrence is real – After someone recovers from an episode of TTP, concerns linger about life-threatening recurrences. Laboratory testing can be incredibly reassuring.

The smallest symptom may trigger a concern. There is often no obvious sign that an episode is going to happen; one minute you are fine and the next you are critically ill. If a patient with TTP has subtle neurologic changes, checking the blood count is the only way to know whether they will crash in the next few hours or if it's a run-of-the-mill headache.