Immune thrombocytopenia- ASH 2019 guidelines

• In adults with a platelet count of ≥20 × 109/L who are asymptomatic or have minor mucocutaneous bleeding, the ASH guideline panel suggests management as an outpatient rather than hospital admission (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯). Remark: Patients who are refractory to treatment, with social concerns, uncertainty about the diagnosis, significant comorbidities with risk of bleeding, and more significant mucosal bleeding may benefit from admission to the hospital. Patients not admitted to the hospital should receive education and expedited follow-up with a hematologist. The need for admission is also variable across the range of platelet counts represented here (20 × 109/L to 150 × 109/L).

3. Duration and type of corticosteroids

• In adults with newly diagnosed ITP, the ASH guideline panel recommends against a prolonged course (>6 weeks including treatment and taper) of prednisone and in favor of a short course (≤6 weeks) (strong recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).


• In adults with newly diagnosed ITP, the ASH guideline panel suggests either prednisone (0.5-2.0 mg/kg per day) or dexamethasone (40 mg per day for 4 days) as the type of corticosteroid for initial therapy (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯). Remark: If a high value is placed on rapidity of platelet count response, an initial course of dexamethasone may be preferred over prednisone, given that dexamethasone showed increased desirable effects with regard to response at 7 days.

• In adults with newly diagnosed ITP, the ASH guideline panel suggests corticosteroids alone rather than rituximab and corticosteroids for initial therapy (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯). Remark: If high value is placed on the possibility for remission over concerns for potential side effects of rituximab, then an initial course of corticosteroids with rituximab may be preferred.

5. Eltrombopag vs romiplostim

• In adults with ITP for ≥3 months who are corticosteroid-dependent or unresponsive to corticosteroids and are going to be treated with a thrombopoietin receptor agonist (TPO-RA), the ASH guideline panel suggests either eltrombopag or romiplostim (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯). Remark: Individual patient preference may place a higher value on the use of a daily oral medication or weekly subcutaneous injections.

6. Second-line therapies: splenectomy, TPO-RA, and rituximab compared 1 against the other.

• In adults with ITP lasting ≥3 months who are corticosteroid-dependent or have no response to corticosteroids, the ASH guideline panel suggests either splenectomy or a TPO-RA (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).In adults with ITP lasting ≥3 months who are corticosteroid-dependent or have no response to corticosteroids, the ASH guideline panel suggests rituximab rather than splenectomy (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).


• In adults with ITP lasting ≥3 months who are corticosteroid-dependent or have no response to corticosteroids, the ASH guideline panel suggests a TPO-RA rather than rituximab (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯). Remark: These recommendations are the result of dichotomousevaluation of treatments that are often being considered simultaneously. Each of these second-line treatments may be effective therapy and therefore the choice of treatment should be individualized based on duration of ITP, frequency of bleeding episodes requiring hospitalization or rescue medication, comorbidities, age of the patient, medication adherence, medical and social support networks, patient values and preferences, cost, and availability. Patient education and shared decision-making are encouraged. If possible, splenectomy should be delayed for at least 1 year after diagnosis because of the potential for spontaneous remission in the first year. Patients who value avoidance of long-term medication may prefer splenectomy or rituximab. Patients who wish to avoid surgery may prefer a TPO-RA or rituximab. Patients who place a high value on achieving a durable response may prefer splenectomy or TPO-RAs.

7. Outpatient vs inpatient management.

• In children with newly diagnosed ITP and a platelet count of <20 × 109/L who have no or mild bleeding (skin manifestations) only, the ASH guideline panel suggests against admission to the hospital and in favor of management as an outpatient (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯). Remark: For patients with uncertainty about the diagnosis, those with social concerns, those who live far from the hospital, and those for whom follow-up cannot be guaranteed, admission to the hospital may be preferable.

8. Treatment vs observation

• In children with newly diagnosed ITP who have no or minor bleeding, the ASH guideline panel suggests observation rather than corticosteroids (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).


• In children with newly diagnosed ITP who have no or minor bleeding, the ASH guideline panel recommends observation rather than IV immunoglobulin (IVIG) (strong recommendation based on moderate certainty in the evidence of effects ⊕⊕⊕◯).


• In children with newly diagnosed ITP who have no or minor bleeding, the ASH guideline panel recommends observation rather than anti-D immunoglobulin (strong recommendation based on moderate certainty in the evidence of effects ⊕⊕⊕◯).

• In children with newly diagnosed ITP who have non–life-threatening mucosal bleeding and/or diminished HRQoL, the ASH guideline panel recommends against courses of corticosteroids longer than 7 days and in favor of courses 7 days or shorter (strong recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).


• In children with newly diagnosed ITP who have non–life-threatening mucosal bleeding and/or diminished HRQoL, the ASH guideline panel suggests prednisone (2-4 mg/kg per day; maximum, 120 mg daily, for 5-7 days) rather than dexamethasone (0.6 mg/kg per day; maximum, 40 mg/kg per day, for 4 days) (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).

10. Treatment of children with non–life-threatening bleeding and/or diminished HRQoL.

• In children with newly diagnosed ITP who have non–life-threatening mucosal bleeding and/or diminished HRQoL, the ASH guideline panel suggests corticosteroids rather than anti-D immunoglobulin (conditional recommendation based on low certainty in the evidence of effects ⊕⊕◯◯). Remark: This recommendation assumes corticosteroid dosing as outlined recommendations 14 and 15. This recommendation is reserved only for children with nonmajor mucosal bleeding.


• In children with newly diagnosed ITP who have non–life-threatening mucosal bleeding and/or diminished HRQoL, the ASH guideline panel suggests either anti-D immunoglobulin or IVIG (conditional recommendation based on low certainty in the evidence of effects ⊕⊕◯◯). Remark: This recommendation is reserved only for children with nonmajor mucosal bleeding.


• In children with newly diagnosed ITP who have non–life-threatening mucosal bleeding and/or diminished HRQoL, the ASH guideline panel suggests corticosteroids rather than IVIG (conditional recommendation based on low certainty in the evidence of effects ⊕⊕◯◯). Remark: This recommendation assumes that a short course of corticosteroids is being used for treatment as recommended in recommendation 14. This recommendation is reserved only for children with nonmajor mucosal bleeding.

• In children with ITP who have non–life-threatening mucosal bleeding and/or diminished HRQoL and do not respond to first-line treatment, the ASH guideline panel suggests rituximab rather than splenectomy (conditional recommendation based on very low certainty in the evidence of effects ⊕◯◯◯).