Risks With Untreated ITP

James B. Bussel, MD:Given all the drawbacks that there can be with ITP treatment, especially with prednisone, what would have happened if she had been left alone and not treated? Typically, even if it’s not well recognized even by the patient who thinks, “Oh yeah, I feel lousy every day but that’s just me,” or by the physician who doesn’t think of ITP as associated with impaired health-related quality of life, these effects can be important in an untreated patient, completely separately from any issues of what is the platelet count and what is the risk of bleeding.

Depending on a given count as to how much bleeding she has and what else is going on, it may make it socially difficult for her to go to work or see people outside the house if her menses are very heavy for the week or so that that may be occurring. If she takes, as many people would recommend, oral contraceptives, if she uses those that contain estrogen, there are women in whom that will make their ITP worse. And estrogen is well known to foster autoimmune disease. On the other hand, she could use just progesterone either with daily Provera (medroxyprogesterone acetate) or Depo-Provera shots.

Although these would work in general to stop her heavy menses, she may have mild intermittent bleeding, and many people do not like the effects of that, especially over time. So, this is also not an optimal approach. In that sense, increasing the platelet count will usually be the best way to reduce heavy menses if that’s a problem, which it sounds as if it may be in her. Other options such as using Amicar, or epsilon aminocaproic acid, or Cyklokapron, which inhibit fibrinolysis, may also lessen the periods. But ITP itself is a prothrombotic disease, at least mildly, so there is an extra risk of clotting when these are used, as there may be with oral contraceptive pills.

So, it gets to be a complicated issue. If the patient feels good, if the heavy menses—as in this case—are not severe, and if the patient prefers no treatment and is not otherwise having important bleeding and no risks of either her activities, her access to medical care, etc, then it may be OK to leave her without treatment for a while and see if she gets better on her own. But almost everybody with platelets less than 30,000, and especially less than 20,000, is going to require treatment usually starting at diagnosis.

Transcript edited for clarity.

Case: A 44-year-old woman presenting with reddish-purple rash on lower legs

February 2017

• Patient presents with complaints of a reddish-purple rash on her lower legs and “constant” bruises appearing “spontaneously” without her remembering any trauma
• Physical evaluation reveals:
  • The rash to be petechiae (subcutaneous bleeding)
  • Slightly overweight (BMI = 26.5 kg/m²)
  • Patient is afebrile, with no splenomegaly
• When asked, reports her menstrual flow is unusually heavy, but says she was evaluated for and had no evidence of fibroids or endometriosis
• No personal or family history of cancer; no recent viral illnesses; no bone pain
• Current medications: no chronic medications; acetaminophen as needed; multivitamin
• Laboratory findings:
  • CBC reveals platelets 21 X 10⁹/L
  • All other findings with normal range
  • Negative forH pylori, HIV, and HCV
• Diagnosis: chronic ITP
  • Started course of prednisone 1 mg/kg X 21 days, then tapered off; at evaluation, platelets: 27 X 10⁹/L
  • Second course of prednisone 1 mg/kg X 21 days; at evaluation, platelets still <30
  • Third course of prednisone 1 mg/kg X 21 days; at evaluation, platelets still <30

February 2018

• &ldquo;Rash&rdquo; partly resolved, bruising still present
• Patient complains of weight gain on treatment and trouble sleeping
• After discussion with patient, she is started on eltrombopag (PROMACTA), at a dose of 50 mg/day