Myelodysplastic syndrome

Use of this site is subject to you reading and agreeing with the terms set forth in the disclaimer.

Is there a regimen missing from this list? Would you like to share a different dosage/schedule or an additional reference for a regimen? Have you noticed an error? Do you have an idea that will help the site grow to better meet your needs and the needs of many others? You are invited to contribute to the site.

24 regimens on this page 43 variants on this page

Untreated

Alemtuzumab (Campath)

back to top

Indication: Intermediate-1 MDS (RAEB-I, RA, or RARS)

Regimen

Phase II

• Alemtuzumab (Campath) 1 mg IV once on day 1; then 10 mg IV once per day on days 2 to 11

Supportive Medications:

• Pentamidine (Nebupent) dose not specified INH monthly for at least 6 months for PCP prophylaxis
• Valacyclovir (Valtrex) dose/schedule not specified until CD4 count >200/uL
• Ciprofloxacin (Cipro) dose/schedule not specified if ANC <500/uL
• Erythropoietin and G-CSF were permitted for severe anemia or neutropenia

11-day course of therapy

References

1. Sloand EM, Olnes MJ, Shenoy A, Weinstein B, Boss C, Loeliger K, Wu CO, More K, Barrett AJ, Scheinberg P, Young NS. Alemtuzumab treatment of intermediate-1 myelodysplasia patients is associated with sustained improvement in blood counts and cytogenetic remissions. J Clin Oncol. 2010 Dec 10;28(35):5166-73. Epub 2010 Nov 1. link to original article contains verified protocol PubMed

Antithymocyte globulin (ATG)

back to top

Regimen

Phase II

• Antithymocyte globulin (ATG) 40 mg/kg IV over 4 to 8 hours on days 1 to 4
• Prednisone (Sterapred) 1 mg/kg (minimum of 40 mg) PO total dose on days 1 to 10, and then tapered to off during days 11 to 17

References

1. Molldrem JJ, Caples M, Mavroudis D, Plante M, Young NS, Barrett AJ. Antithymocyte globulin for patients with myelodysplastic syndrome. Br J Haematol. 1997 Dec;99(3):699-705. PubMed
2. Molldrem JJ, Leifer E, Bahceci E, Saunthararajah Y, Rivera M, Dunbar C, Liu J, Nakamura R, Young NS, Barrett AJ. Antithymocyte globulin for treatment of the bone marrow failure associated with myelodysplastic syndromes. Ann Intern Med. 2002 Aug 6;137(3):156-63. link to original article contains protocol PubMed
3. Steensma DP, Dispenzieri A, Moore SB, Schroeder G, Tefferi A. Antithymocyte globulin has limited efficacy and substantial toxicity in unselected anemic patients with myelodysplastic syndrome. Blood. 2003 Mar 15;101(6):2156-8. Epub 2002 Oct 31. link to original article contains protocol PubMed
4. Sloand EM, Wu CO, Greenberg P, Young N, Barrett J. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol. 2008 May 20;26(15):2505-11. Epub 2008 Apr 14. link to original article PubMed

Azacitidine (Vidaza)

back to top

Regimen #1, Silverman et al. 2002 & 2006 (CALGB 8421, 8921, & 9921)

Phase III

• Azacitidine (Vidaza) 75 mg/m2 SC or IV continuous infusion on days 1 to 7

28-day cycles, given for at least 4 cycles, then depending on study, continued for 3 cycles beyond complete remission, or if there was progressive disease or unacceptable toxicity

Regimen #2, Fili et al. 2013

Phase II

Intended to be used for low-risk MDS patients who are symptomatic or intolerant to erythropoietin

• Azacitidine (Vidaza) 75 mg/m2 SC once per day on days 1 to 5

Supportive medications:

• G-CSF or GM-CSF was allowed if ANC < 200 and/or systemic infection
• Erythropoiesis-stimulating agents were not allowed
• Antimicrobial and antifungal prophylaxis (agents not specified) given if ANC < 500

28-day cycles x 8 cycles

Regimen #3, Grövdal et al. 2010

Phase II

Intended to be used for high-risk MDS patients in remission after induction therapy

• Azacitidine (Vidaza) 60 mg/m2 SC once per day on days 1 to 5

28-day cycles

References

1. Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002 May 15;20(10):2429-40. link to original article contains verified protocol PubMed
   1. Update: Silverman LR, McKenzie DR, Peterson BL, Holland JF, Backstrom JT, Beach CL, Larson RA; Cancer and Leukemia Group B. Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J Clin Oncol. 2006 Aug 20;24(24):3895-903. link to original article contains protocol PubMed
2. Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. Epub 2009 Feb 21. link to original article contains protocol PubMed
3. Grövdal M, Karimi M, Khan R, Aggerholm A, Antunovic P, Astermark J, Bernell P, Engström LM, Kjeldsen L, Linder O, Nilsson L, Olsson A, Holm MS, Tangen JM, Wallvik J, Oberg G, Hokland P, Jacobsen SE, Porwit A, Hellström-Lindberg E. Maintenance treatment with azacytidine for patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukaemia following MDS in complete remission after induction chemotherapy. Br J Haematol. 2010 Aug;150(3):293-302. Epub 2010 May 20. link to original article contains verified protocol PubMed
4. Filì C, Malagola M, Follo MY, Finelli C, Iacobucci I, Martinelli G, Cattina F, Clissa C, Candoni A, Fanin R, Gobbi M, Bocchia M, Defina M, Spedini P, Skert C, Manzoli L, Cocco L, Russo D. Prospective phase II Study on 5-days azacitidine for treatment of symptomatic and/or erythropoietin unresponsive patients with low/INT-1-risk myelodysplastic syndromes. Clin Cancer Res. 2013 Jun 15;19(12):3297-308. link to original article contains verified protocol PubMed

Azacitidine & Lenalidomide

back to top

Regimen

Phase II

• Azacitidine (Vidaza) 75 mg/m2 IV once per day on days 1 to 5
• Lenalidomide (Revlimid) 10mg PO once per day on days 1 to 21

28-day cycles up to 7 cycles, with option to continue single agent azacitidine per MD discretion

References

1. Sekeres MA, Tiu RV, Komrokji R, Lancet J, Advani AS, Afable M, Englehaupt R, Juersivich J, Cuthbertson D, Paleveda J, Tabarroki A, Visconte V, Makishima H, Jerez A, Paquette R, List AF, Maciejewski JP. Phase 2 study of the lenalidomide and azacitidine combination in patients with higher-risk myelodysplastic syndromes. Blood. 2012 Dec 13;120(25):4945-51. Epub 2012 Aug 22. link to original article contains protocol PubMed

Azacitidine & Vorinostat

back to top

Regimen

Phase II

Three different schedules were evaluated; a randomized trial is underway using the doses from schedule two.

Schedule One

• Azacitidine (Vidaza) 55 mg/m2 SC once per day on days 1 to 7
• Vorinostat (Zolinza) 400 mg PO once per day on days 3 to 16

Schedule Two

• Azacitidine (Vidaza) 75 mg/m2 SC once per day on days 1 to 7
• Vorinostat (Zolinza) 600 mg PO once per day on days 3 to 9

Schedule Three

• Azacitidine (Vidaza) 55 mg/m2 SC once per day on days 1 to 7
• Vorinostat (Zolinza) 400 mg PO once per day on days 3 to 9

28-day cycles

References

1. Abstract: Amit Verma, Rosalie Odchimar-Reissig, Eric J. Feldman, Shyamala C. Navada, Erin P Demakos, Maria R. Baer, Vesna Najfeld, Joseph A Sparano, Richard Piekarz. A Phase II Trial Of Epigenetic Modulators Vorinostat In Combination With Azacitidine (azaC) In Patients With The Myelodysplastic Syndrome (MDS): Initial Results Of Study 6898 Of The New York Cancer Consortium. Blood Nov 2013,122(21)386 link to original abstract

Best supportive care

back to top

Regimen

Phase III

No active antineoplastic therapy; included here because it was a comparator arm in several studies.

References

1. Silverman LR, Demakos EP, Peterson BL, Kornblith AB, Holland JC, Odchimar-Reissig R, Stone RM, Nelson D, Powell BL, DeCastro CM, Ellerton J, Larson RA, Schiffer CA, Holland JF. Randomized controlled trial of azacitidine in patients with the myelodysplastic syndrome: a study of the cancer and leukemia group B. J Clin Oncol. 2002 May 15;20(10):2429-40. link to original article contains verified protocol PubMed
   1. Update: Silverman LR, McKenzie DR, Peterson BL, Holland JF, Backstrom JT, Beach CL, Larson RA; Cancer and Leukemia Group B. Further analysis of trials with azacitidine in patients with myelodysplastic syndrome: studies 8421, 8921, and 9221 by the Cancer and Leukemia Group B. J Clin Oncol. 2006 Aug 20;24(24):3895-903. link to original article contains protocol PubMed
2. Kantarjian H, Issa JP, Rosenfeld CS, Bennett JM, Albitar M, DiPersio J, Klimek V, Slack J, de Castro C, Ravandi F, Helmer R 3rd, Shen L, Nimer SD, Leavitt R, Raza A, Saba H. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer. 2006 Apr 15;106(8):1794-803. link to original article contains protocol PubMed
3. Fenaux P, Mufti GJ, Hellstrom-Lindberg E, Santini V, Finelli C, Giagounidis A, Schoch R, Gattermann N, Sanz G, List A, Gore SD, Seymour JF, Bennett JM, Byrd J, Backstrom J, Zimmerman L, McKenzie D, Beach C, Silverman LR; International Vidaza High-Risk MDS Survival Study Group. Efficacy of azacitidine compared with that of conventional care regimens in the treatment of higher-risk myelodysplastic syndromes: a randomised, open-label, phase III study. Lancet Oncol. 2009 Mar;10(3):223-32. Epub 2009 Feb 21. link to original article contains protocol PubMed content property of HemOnc.org
4. Lübbert M, Suciu S, Baila L, Rüter BH, Platzbecker U, Giagounidis A, Selleslag D, Labar B, Germing U, Salih HR, Beeldens F, Muus P, Pflüger KH, Coens C, Hagemeijer A, Eckart Schaefer H, Ganser A, Aul C, de Witte T, Wijermans PW. Low-dose decitabine versus best supportive care in elderly patients with intermediate- or high-risk myelodysplastic syndrome (MDS) ineligible for intensive chemotherapy: final results of the randomized phase III study of the European Organisation for Research and Treatment of Cancer Leukemia Group and the German MDS Study Group. J Clin Oncol. 2011 May 20;29(15):1987-96. Epub 2011 Apr 11. link to original article contains verified protocol PubMed

Clofarabine (Clolar)

back to top

Regimen #1, Faderl et al. 2010

Non-randomized

Initial dose was too toxic; 20 mg/m2 was final dose level

• Clofarabine (Clolar) 20 mg/m2 PO once per day on days 1 to 5

Supportive medications:

• "Supportive care included anti-infectious prophylaxis (eg, levaquin, valacyclovir, and itraconazole or voriconazole), hematopoietic growth factors, and transfusions as judged indicated by the treating physician." In order to decrease risk of liver function abnormalities, no antifungals were given on the days where clofarabine was given.

4 to 8 week cycles x up to 12 cycles

Regimen #2, Faderl et al. 2012

Randomized Phase II, >20 per arm

This randomized trial tested two doses of clofarabine, 15 mg/m2 and 30 mg/m2. Lower dose was less toxic; clinical activity was comparable

• Clofarabine (Clolar) 15 mg/m2 or 30 mg/m2 IV over 1 hour once per day on days 1 to 5

Supportive medications:

• "Supportive care measures such as antibiotic prophylaxis (eg, levofloxacin, valacyclovir, and itraconazole or voriconazole), hematopoietic growth factors, and transfusions were provided as necessitated for optimal medical care of the patients." In order to decrease risk of liver function abnormalities, no antifungals were given on the days where clofarabine was given.

4 to 8 week cycles x up to 12 cycles

References

1. Faderl S, Garcia-Manero G, Estrov Z, Ravandi F, Borthakur G, Cortes JE, O'Brien S, Gandhi V, Plunkett W, Byrd A, Kwari M, Kantarjian HM. Oral clofarabine in the treatment of patients with higher-risk myelodysplastic syndrome. J Clin Oncol. 2010 Jun 1;28(16):2755-60. Epub 2010 Apr 26. link to original article contains verified protocol PubMed
2. Faderl S, Garcia-Manero G, Jabbour E, Ravandi F, Borthakur G, Estrov Z, Gandhi V, Byrd AL, Kwari M, Cortes J, Kantarjian HM. A randomized study of 2 dose levels of intravenous clofarabine in the treatment of patients with higher-risk myelodysplastic syndrome. Cancer. 2012 Feb 1;118(3):722-8. Epub 2011 Jul 12. link to original article contains verified protocol PubMed

Cyclosporine modified (Neoral)

back to top

Regimen

Pilot, <20 patients reported

• Cyclosporine modified (Neoral) 5 to 6 mg/kg/day, divided into two equal doses PO BID, adjusted to maintain therapeutic cyclosporine level of 100 to 300 ng/mL

References

1. Jonásova A, Neuwirtová R, Cermák J, Vozobulová V, Mociková K, Sisková M, Hochová I. Cyclosporin A therapy in hypoplastic MDS patients and certain refractory anaemias without hypoplastic bone marrow. Br J Haematol. 1998 Feb;100(2):304-9. link to original article contains protocol PubMed
2. Sloand EM, Wu CO, Greenberg P, Young N, Barrett J. Factors affecting response and survival in patients with myelodysplasia treated with immunosuppressive therapy. J Clin Oncol. 2008 May 20;26(15):2505-11. Epub 2008 Apr 14. link to original article PubMed

Decitabine (Dacogen)

back to top

Regimen #1, Kantarjian et al. 2006

Randomized Phase II, >20 per arm

• Decitabine (Dacogen) 20 mg/m2 IV over 1 hour once per day on days 1 to 5

28-day cycles

Regimen #2, Kantarjian et al. 2006; Rüter et al. 2006, Lübbert et al. 2011

Phase III

• Decitabine (Dacogen) 15 mg/m2 IV over 3 to 4 hours every 8 hours on days 1 to 3

6-week cycles x up to 10 cycles, depending on the protocol

Regimen #3, Kantarjian et al. 2006

Randomized Phase II, <20 in this arm

• Decitabine (Dacogen) 20 mg/m2/day divided into 2 SC doses per day on days 1 to 5

28-day cycles

Regimen #4, Kantarjian et al. 2006

Randomized Phase II, <20 in this arm

• Decitabine (Dacogen) 10 mg/m2 IV over 1 hour once per day on days 1 to 10

28-day cycles

Regimen #5, Garcia-Manero et al. 2013

Phase II

• Decitabine (Dacogen) 20 mg/m2 SC once per day on days 1 to 3

28-day cycles +/- 3 days x up to 12 months

Regimen #6, Garcia-Manero et al. 2013

Phase II

• Decitabine (Dacogen) 20 mg/m2 SC once per week on days 1, 8, 15

28-day cycles +/- 3 days x up to 12 months

References

1. Rüter B, Wijermans PW, Lübbert M. Superiority of prolonged low-dose azanucleoside administration? Results of 5-aza-2'-deoxycytidine retreatment in high-risk myelodysplasia patients. Cancer. 2006 Apr 15;106(8):1744-50. link to original article contains protocol PubMed
2. Kantarjian H, Issa JP, Rosenfeld CS, Bennett JM, Albitar M, DiPersio J, Klimek V, Slack J, de Castro C, Ravandi F, Helmer R 3rd, Shen L, Nimer SD, Leavitt R, Raza A, Saba H. Decitabine improves patient outcomes in myelodysplastic syndromes: results of a phase III randomized study. Cancer. 2006 Apr 15;106(8):1794-803. link to original article contains protocol PubMed
3. Kantarjian H, Oki Y, Garcia-Manero G, Huang X, O'Brien S, Cortes J, Faderl S, Bueso-Ramos C, Ravandi F, Estrov Z, Ferrajoli A, Wierda W, Shan J, Davis J, Giles F, Saba HI, Issa JP. Results of a randomized study of 3 schedules of low-dose decitabine in higher-risk myelodysplastic syndrome and chronic myelomonocytic leukemia. Blood. 2007 Jan 1;109(1):52-7. Epub 2006 Aug 1. link to original article contains protocol PubMed
4. Lübbert M, Suciu S, Baila L, Rüter BH, Platzbecker U, Giagounidis A, Selleslag D, Labar B, Germing U, Salih HR, Beeldens F, Muus P, Pflüger KH, Coens C, Hagemeijer A, Eckart Schaefer H, Ganser A, Aul C, de Witte T, Wijermans PW. Low-dose decitabine versus best supportive care in elderly patients with intermediate- or high-risk myelodysplastic syndrome (MDS) ineligible for intensive chemotherapy: final results of the randomized phase III study of the European Organisation for Research and Treatment of Cancer Leukemia Group and the German MDS Study Group. J Clin Oncol. 2011 May 20;29(15):1987-96. Epub 2011 Apr 11. link to original article contains verified protocol PubMed
5. Garcia-Manero G, Jabbour E, Borthakur G, Faderl S, Estrov Z, Yang H, Maddipoti S, Godley LA, Gabrail N, Berdeja JG, Nadeem A, Kassalow L, Kantarjian H. Randomized Open-Label Phase II Study of Decitabine in Patients With Low- or Intermediate-Risk Myelodysplastic Syndromes. J Clin Oncol. 2013 Jun 3. [Epub ahead of print] link to original article contains verified protocol PubMed

Epoetin alfa & Lenalidomide

back to top

Phase II

Regimen

• Lenalidomide (Revlimid) 10 to 15 mg PO once per day

For 16 weeks; erythroid nonresponders or those with relapsed anemia then offered combined treatment:

• Epoetin alfa (Procrit) 40,000 units SC once per week
• Lenalidomide (Revlimid) 10 to 15 mg PO once per day

Continue until treatment failure or limiting toxicity

References

1. Komrokji RS, Lancet JE, Swern AS, Chen N, Paleveda J, Lush R, Saba HI, List AF. Combined treatment with lenalidomide and epoetin alfa in lower-risk patients with myelodysplastic syndrome. Blood. 2012 Oct 25;120(17):3419-24. Epub 2012 Aug 30. link to original article contains protocol PubMed

Lenalidomide (Revlimid)

back to top

Regimen

Phase II

• Lenalidomide (Revlimid) 10 mg PO once per day

References

1. List A, Kurtin S, Roe DJ, Buresh A, Mahadevan D, Fuchs D, Rimsza L, Heaton R, Knight R, Zeldis JB. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med. 2005 Feb 10;352(6):549-57. link to original article PubMed
2. List A, Dewald G, Bennett J, Giagounidis A, Raza A, Feldman E, Powell B, Greenberg P, Thomas D, Stone R, Reeder C, Wride K, Patin J, Schmidt M, Zeldis J, Knight R; Myelodysplastic Syndrome-003 Study Investigators. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med. 2006 Oct 5;355(14):1456-65. link to original article contains protocol PubMed
3. Raza A, Reeves JA, Feldman EJ, Dewald GW, Bennett JM, Deeg HJ, Dreisbach L, Schiffer CA, Stone RM, Greenberg PL, Curtin PT, Klimek VM, Shammo JM, Thomas D, Knight RD, Schmidt M, Wride K, Zeldis JB, List AF. Phase 2 study of lenalidomide in transfusion-dependent, low-risk, and intermediate-1 risk myelodysplastic syndromes with karyotypes other than deletion 5q. Blood. 2008 Jan 1;111(1):86-93. Epub 2007 Sep 24. link to original article contains protocol PubMed

Rabbit ATG (Thymoglobulin)

back to top

Regimen

Phase II

• Rabbit ATG (Thymoglobulin) 2.5 mg/kg IV over at least 6 hours once per day on days 1 to 4

Supportive medications:

• Prednisone (Sterapred) 1 mg/kg/day PO, started 2 days before first rabbit ATG (Thymoglobulin) and continued at full dose during the 4 days, then tapered over the subsequent 14 days (tapering schedule not described)
• Antibiotics per local practices

References

1. Komrokji RS, Mailloux AW, Chen DT, Sekeres MA, Paquette R, Fulp WJ, Sugimori C, Paleveda-Pena J, Maciejewski JP, List AF, Epling-Burnette PK. A phase 2 multicenter rabbit anti-thymocyte globulin trial in patients withmyelodysplastic syndromes identifying a novel model for responseprediction. Haematologica. 2014 Jan 31. [Epub ahead of print] link to original article contains verified protocol PubMed

Temozolomide (Temodar)

back to top

Regimen, Brandwein et al. 2014

Phase II

Patient selection was based on MGMT expression by Western blot. See article for details.

• Temozolomide (Temodar) 200 mg/m2/day PO on days 1 to 7; complete responders could receive 200 mg/m2/day PO on days 1 to 5

28-day cycle x up to 12 cycles

References

1. Brandwein JM, Kassis J, Leber B, Hogge D, Howson-Jan K, Minden MD, Galarneau A, Pouliot JF. Phase II study of targeted therapy with temozolomide in acute myeloid leukaemia and high-risk myelodysplastic syndrome patients pre-screened for low O(6) -methylguanine DNA methyltransferase expression. Br J Haematol. 2014 Dec;167(5):664-70. Epub 2014 Aug 27. link to original article contains protocol PubMed

Links

• IPSS-R calculator, Revised International Prognostic Scoring System calculator (mds-foundation.org)
• Advanced IPSS-R calculator, Revised International Prognostic Scoring System calculator (mds-foundation.org)