Pharmacologic therapies for symptom control in adult patients with indolent systemic mastocytosis
| Symptom . | Treatment ladder* . | Drug class . | Specific drugs/doses . | Common side effects (>5%-10%) and precautions† . |
|---|---|---|---|---|
| Pruritus, flushing | First line | H1-antagonist | Cetirizine, 5-10 mg/d‡ | Headache, somnolence, confusion, asthenia, xerostomia |
| Fexofenadine, 60 mg BID or 180 mg/d‡ | Precautions: Hydroxyzine-anti-cholinergic effects: use with caution in older patients, those with glaucoma, BPH, asthma, and similar conditions | |||
| Hydroxyzine, 25 mg q 6 h‡ | ||||
| *Doses can be increased with supervision if indicated | ||||
| Second line | Leukotriene antagonist | Montelukast, 10 mg/d | Headache Precautions: liver function impairment, neuropsychiatric conditions | |
| Zafirlukast, 20 mg BID | ||||
| Third line | Nonsteroidal anti-inflammatory drug | Aspirin (see text) | Gastrointestinal bleeding, peptic ulcer disease Precautions: may precipitate anaphylactic reaction (see text), aspirin hypersensitivity, children/adolescents with flu (Reye’s syndrome), hepatic or renal dysfunction, bleeding disorders | |
| Third line | Psolaren plus UV A photochemotherapy | See specialized texts | Nausea, pruritus, erythema of varying degree, increased risk of nonmelanoma skin cancers | |
| Contraindications: Pregnancy, xeroderma pigmentosa, lupus erythematosus with photosensitivity | ||||
| Abdominal pain, cramping, diarrhea, heartburn, nausea, vomiting | First line | H2-antagonist | Ranitidine, 150 mg BID | Headache, abdominal pain, dizziness, constipation, diarrhea |
| Famotidine, 10 mg BID | Cimetidine: gynecomastia | |||
| Cimetidine, 400 mg BID | ||||
| Second line | Proton pump inhibitor | Omeprazole, 20 mg/d | Headache, abdominal pain, nausea, vomiting, diarrhea, flatulence | |
| Pantoprazole, 40 mg/d | ||||
| Rabeprazole, 20 mg/d | ||||
| Third line | Sodium cromolyn | 100-200 mg QID 30 min before meals and bedtime | Dysgeusia, cough, osmotic diarrhea | |
| Fourth line | Corticosteroid | Prednisone, 0.5-1 mg/kg/d starting dose; taper as feasible based on response/tolerance | Dose/duration dependent (consult comprehensive drug reference resource) | |
| Headache, cognitive impairment, depression | First line | H1- and H2-antagonist | As above | As above |
| Second line | Sodium cromolyn | As above | As above | |
| Recurrent hypotension∗ | First line | Epinephrine | See text | See text |
| Second line | H1- and H2-antagonists | As above | As above | |
| Third line | Corticosteroid | Prednisone (as above) | As above | |
| Fourth line | Cytoreductive therapy (IFN-α or 2-chlorodeoxyadenosine) | See text/below | See text/below | |
| Osteoporosis | First line | Bisphosphonate | Alendronate, 70 mg q wk | Flu-like symptoms, abdominal pain, nausea, vomiting, diarrhea, asthenia, hypocalcemia, rash, musculoskeletal pain, headache, osteonecrosis of the jaw, nephrotoxicity Follow established guidelines for bisphosphonate use (see text) |
| Risedronate, 35 mg q week | Precautions: esophageal/upper GI disease (oral bisphosphonates), renal disease, poor oral hygiene or dental procedures | |||
| Pamidronic acid, 90 mg IV q 4 wk | ||||
| Zoledronic acid, 4 mg IV q 4 wk | ||||
| Second line | Cytokine/ immunomodulatory drug | IFN-α, starting dose, 1-3 MU SQ 3 × per wk; target dose, 3-5 MU SQ 3–5 × per wk | Dose dependent (consult comprehensive drug reference resource) Comment: pegylated interferon may be better tolerated | |
| Third line | Purine nucleoside analog | 2-chlorodeoxyadenosine (cladribine/2-CdA), 5 mg/m2 IV × 5 d every 4-8 wk | Myelosuppression, immunosuppression |
| Symptom . | Treatment ladder* . | Drug class . | Specific drugs/doses . | Common side effects (>5%-10%) and precautions† . |
|---|---|---|---|---|
| Pruritus, flushing | First line | H1-antagonist | Cetirizine, 5-10 mg/d‡ | Headache, somnolence, confusion, asthenia, xerostomia |
| Fexofenadine, 60 mg BID or 180 mg/d‡ | Precautions: Hydroxyzine-anti-cholinergic effects: use with caution in older patients, those with glaucoma, BPH, asthma, and similar conditions | |||
| Hydroxyzine, 25 mg q 6 h‡ | ||||
| *Doses can be increased with supervision if indicated | ||||
| Second line | Leukotriene antagonist | Montelukast, 10 mg/d | Headache Precautions: liver function impairment, neuropsychiatric conditions | |
| Zafirlukast, 20 mg BID | ||||
| Third line | Nonsteroidal anti-inflammatory drug | Aspirin (see text) | Gastrointestinal bleeding, peptic ulcer disease Precautions: may precipitate anaphylactic reaction (see text), aspirin hypersensitivity, children/adolescents with flu (Reye’s syndrome), hepatic or renal dysfunction, bleeding disorders | |
| Third line | Psolaren plus UV A photochemotherapy | See specialized texts | Nausea, pruritus, erythema of varying degree, increased risk of nonmelanoma skin cancers | |
| Contraindications: Pregnancy, xeroderma pigmentosa, lupus erythematosus with photosensitivity | ||||
| Abdominal pain, cramping, diarrhea, heartburn, nausea, vomiting | First line | H2-antagonist | Ranitidine, 150 mg BID | Headache, abdominal pain, dizziness, constipation, diarrhea |
| Famotidine, 10 mg BID | Cimetidine: gynecomastia | |||
| Cimetidine, 400 mg BID | ||||
| Second line | Proton pump inhibitor | Omeprazole, 20 mg/d | Headache, abdominal pain, nausea, vomiting, diarrhea, flatulence | |
| Pantoprazole, 40 mg/d | ||||
| Rabeprazole, 20 mg/d | ||||
| Third line | Sodium cromolyn | 100-200 mg QID 30 min before meals and bedtime | Dysgeusia, cough, osmotic diarrhea | |
| Fourth line | Corticosteroid | Prednisone, 0.5-1 mg/kg/d starting dose; taper as feasible based on response/tolerance | Dose/duration dependent (consult comprehensive drug reference resource) | |
| Headache, cognitive impairment, depression | First line | H1- and H2-antagonist | As above | As above |
| Second line | Sodium cromolyn | As above | As above | |
| Recurrent hypotension∗ | First line | Epinephrine | See text | See text |
| Second line | H1- and H2-antagonists | As above | As above | |
| Third line | Corticosteroid | Prednisone (as above) | As above | |
| Fourth line | Cytoreductive therapy (IFN-α or 2-chlorodeoxyadenosine) | See text/below | See text/below | |
| Osteoporosis | First line | Bisphosphonate | Alendronate, 70 mg q wk | Flu-like symptoms, abdominal pain, nausea, vomiting, diarrhea, asthenia, hypocalcemia, rash, musculoskeletal pain, headache, osteonecrosis of the jaw, nephrotoxicity Follow established guidelines for bisphosphonate use (see text) |
| Risedronate, 35 mg q week | Precautions: esophageal/upper GI disease (oral bisphosphonates), renal disease, poor oral hygiene or dental procedures | |||
| Pamidronic acid, 90 mg IV q 4 wk | ||||
| Zoledronic acid, 4 mg IV q 4 wk | ||||
| Second line | Cytokine/ immunomodulatory drug | IFN-α, starting dose, 1-3 MU SQ 3 × per wk; target dose, 3-5 MU SQ 3–5 × per wk | Dose dependent (consult comprehensive drug reference resource) Comment: pegylated interferon may be better tolerated | |
| Third line | Purine nucleoside analog | 2-chlorodeoxyadenosine (cladribine/2-CdA), 5 mg/m2 IV × 5 d every 4-8 wk | Myelosuppression, immunosuppression |
Treatments can be combined in a stepwise manner for inadequate symptom control at prior step if clinically indicated and feasible.
Basic overview provided. Consult a comprehensive drug reference manual for detailed information regarding feasibility of use during pregnancy, black box warnings, specific contraindications and precautions, drug–drug interactions, dose reduction for hepatic and renal dysfunction, and so on.
Voidance of symptom trigger(s) applies to all patients. Those at risk of anaphylaxis should carry an emergency kit with self-injected epinephrine (EpiPen) (see text). Immunotherapy can be considered in those with IgE-mediated allergic reactions (see text).
BID, twice a day; BPH, benign prostatic hypertrophy; GI, gastrointestinal; H1- and H2-, histamine receptor 1 and 2, respectively; MU, million units; q, every; QD, once daily; QID, four times a day; SQ, subcutaneously; IV, intravenously.