Pheochromocytoma

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3 regimens on this page 5 variants on this page

Guidelines

Given the rapid change in evidence in many areas of hematology/oncology, readers are encouraged to consider any guideline published 5+ years ago to be for historical purposes, only.

ESMO-EURACAN

• 2020: Fassnacht et al. Adrenocortical carcinomas and malignant phaeochromocytomas: ESMO–EURACAN Clinical Practice Guidelines for diagnosis, treatment and follow-up PubMed

NANETS

• 2021: Fishbein et al. The North American Neuroendocrine Tumor Society Consensus Guidelines for Surveillance and Management of Metastatic and/or Unresectable Pheochromocytoma and Paraganglioma PubMed

NCCN

• NCCN does not currently have guidelines at this granular level; please see NCCN Guidelines - Neuroendocrine and Adrenal Tumors.

All lines of therapy

Cyclophosphamide, Dacarbazine, Vincristine

CVD: Cyclophosphamide, Vincristine, Dacarbazine

Regimen

Study	Dates of enrollment	Evidence
Averbuch et al. 1988	1983-08 to 1985-12	Pilot, fewer than 20 pts

Chemotherapy

• Cyclophosphamide (Cytoxan) 750 mg/m² IV once on day 1
• Dacarbazine (DTIC) 600 mg/m² IV once per day on days 1 & 2
• Vincristine (Oncovin) 1.4 mg/m² IV once on day 1

21-day cycles

References

1. Averbuch SD, Steakley CS, Young RC, Gelmann EP, Goldstein DS, Stull R, Keiser HR. Malignant pheochromocytoma: effective treatment with a combination of cyclophosphamide, vincristine, and dacarbazine. Ann Intern Med. 1988 Aug 15;109(4):267-73. link to original article dosing details in abstract have been reviewed by our editors PubMed

Iobenguane I 131 monotherapy

Regimen variant #1

Study	Dates of enrollment	Evidence
Pryma et al. 2018 (MIP-IB12B)	2009-06 to 2016-02	Phase 2 (RT)

Note: This was the study cited by the FDA in their approval.

Radiotherapy

• Iobenguane I 131 (Azedra) by the following weight-based criteria:
  • More than 62.5 kg: 18.5 GBq IV over 30 minutes once on day 1
  • 62.5 kg or less: 0.296 GBq/kg IV over 30 minutes once on day 1

90-day cycle for up to 2 cycles

Regimen variant #2

Study	Dates of enrollment	Evidence
Rose et al. 2003b	1991-2000	Pilot, fewer than 20 pts

Note: Patients underwent stem cell harvest prior to treatment in case autologous stem cell infusion was needed.

Radiotherapy

• Iobenguane I 131 (Azedra) 12 to 18 mCi/kg (maximum single dose of 850 mCi) IV over 2 hours once on day 1

Supportive therapy

• Intravenous fluids started 12 hours before 131I-MIBG administration.
• Potassium iodide (KI) 6 mg/kg PO once at least 2 hours prior to 131I-MIBG, then potassium iodide (KI) 0.88 mg/kg PO Q4H x 7 days, then potassium iodide (KI) 1 mg/kg PO (frequency not specified) "for 45 days after the infusion."
• Potassium perchlorate 8 mg/kg once at least 2 hours prior to 131I-MIBG, then potassium perchlorate 2 mg/kg PO every 6 hours x 5 days.

"Repeat 131I-MIBG treatments were administered on a case-by-case basis in an effort to improve the overall response"

Regimen variant #3

Study	Dates of enrollment	Evidence
Krempf et al. 1991	Not reported in abstract	Pilot, fewer than 20 pts

Radiotherapy

• Iobenguane I 131 (Azedra) 740 megabequerel/mg every 3 months

References

1. Krempf M, Lumbroso J, Mornex R, Brendel AJ, Wemeau JL, Delisle MJ, Aubert B, Carpentier P, Fleury-Goyon MC, Gibold C, Guyot M, Lahneche B, Marchandise X, Schlumberger M, Charbonnel B, Chatal JF. Use of m-[131I]iodobenzylguanidine in the treatment of malignant pheochromocytoma. J Clin Endocrinol Metab. 1991 Feb;72(2):455-61. link to original article dosing details in abstract have been reviewed by our editors PubMed
2. Rose B, Matthay KK, Price D, Huberty J, Klencke B, Norton JA, Fitzgerald PA. High-dose 131I-metaiodobenzylguanidine therapy for 12 patients with malignant pheochromocytoma. Cancer. 2003 Jul 15;98(2):239-48. link to original article dosing details in manuscript have been reviewed by our editors PubMed
3. MIP-IB12B: Pryma DA, Chin BB, Noto RB, Dillon JS, Perkins S, Solnes L, Kostakoglu L, Serafini AN, Pampaloni MH, Jensen J, Armor T, Lin T, White T, Stambler N, Apfel S, DiPippo VA, Mahmood S, Wong V, Jimenez C. Efficacy and Safety of High-Specific-Activity 131I-MIBG Therapy in Patients with Advanced Pheochromocytoma or Paraganglioma. J Nucl Med. 2019 May;60(5):623-630. Epub 2018 Oct 5. link to original article link to PMC article PubMed NCT00874614

Subsequent lines of therapy

Sunitinib monotherapy

Regimen

Study	Dates of enrollment	Evidence	Comparator	Comparative Efficacy
Baudin et al. 2024 (FIRSTMAPPP)	2011-12-01 to 2019-01-31	Randomized Phase 2 (E-esc)	Placebo	Superior PFS12 (primary endpoint) PFS12: 36% vs 19%

Targeted therapy

• Sunitinib (Sutent) 37.5 mg PO once per day on days 1 to 28

28-day cycles

References

1. FIRSTMAPPP: Baudin E, Goichot B, Berruti A, Hadoux J, Moalla S, Laboureau S, Nölting S, de la Fouchardière C, Kienitz T, Deutschbein T, Zovato S, Amar L, Haissaguerre M, Timmers H, Niccoli P, Faggiano A, Angokai M, Lamartina L, Luca F, Cosentini D, Hahner S, Beuschlein F, Attard M, Texier M, Fassnacht M; ENDOCAN-COMETE; ENSAT Networks. Sunitinib for metastatic progressive phaeochromocytomas and paragangliomas: results from FIRSTMAPPP, an academic, multicentre, international, randomised, placebo-controlled, double-blind, phase 2 trial. Lancet. 2024 Mar 16;403(10431):1061-1070. Epub 2024 Feb 22. link to original article dosing details in abstract have been reviewed by our editors PubMed NCT01371201