Difference between revisions of "Paroxysmal nocturnal hemoglobinuria"
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|[[#top|back to top]] | |[[#top|back to top]] | ||
|} | |} | ||
| + | ===Variant #1, 12-week course {{#subobject:4bb05f |Variant=1}}=== | ||
| + | {| class="wikitable" style="width: 100%; text-align:center;" | ||
| + | !Study | ||
| + | ![[Levels_of_Evidence#Evidence|Evidence]] | ||
| + | |- | ||
| + | |[http://www.nejm.org/doi/full/10.1056/NEJMoa031688 Hillmen et al. 2004] | ||
| + | |style="background-color:#ffffbe"|Pilot, <20 pts | ||
| + | |- | ||
| + | |} | ||
| + | ====Therapy==== | ||
| + | *[[Eculizumab (Soliris)]] as follows: | ||
| + | **Weeks 1 to 4: 600 mg IV over 25 to 45 minutes once per week | ||
| + | **Weeks 5 to 12: 900 mg IV over 25 to 45 minutes once every 2 weeks | ||
| − | ===Variant # | + | '''12-week course''' |
| + | |||
| + | ===Variant #2, 6-month course {{#subobject:1 |Variant=1}}=== | ||
{| class="wikitable" style="width: 100%; text-align:center;" | {| class="wikitable" style="width: 100%; text-align:center;" | ||
!Study | !Study | ||
| Line 32: | Line 47: | ||
|- | |- | ||
|[http://www.nejm.org/doi/full/10.1056/NEJMoa061648 Hillmen et al. 2006 (TRIUMPH)] | |[http://www.nejm.org/doi/full/10.1056/NEJMoa061648 Hillmen et al. 2006 (TRIUMPH)] | ||
| − | |style="background-color:#1a9851"|Phase III | + | |style="background-color:#1a9851"|Phase III (E) |
|[[Paroxysmal_nocturnal_hemoglobinuria_(PNH)#Placebo|Placebo]] | |[[Paroxysmal_nocturnal_hemoglobinuria_(PNH)#Placebo|Placebo]] | ||
|style="background-color:#1a9850"|Superior stabilization of Hgb levels and QoL | |style="background-color:#1a9850"|Superior stabilization of Hgb levels and QoL | ||
|- | |- | ||
|} | |} | ||
| − | + | ====Therapy==== | |
| − | |||
| − | |||
| − | ==== | ||
*[[Eculizumab (Soliris)]] as follows: | *[[Eculizumab (Soliris)]] as follows: | ||
| − | **600 mg IV over 25 to 45 minutes once per week | + | **Weeks 1 to 4: 600 mg IV over 25 to 45 minutes once per week |
| − | ** | + | **Weeks 5 to 26: 900 mg IV over 25 to 45 minutes once every 2 weeks |
| − | |||
| − | |||
| − | |||
| − | |||
| − | |||
| − | |||
| − | |||
====Supportive medications==== | ====Supportive medications==== | ||
*Patients received Neisseria meningitidis meningococcal vaccination | *Patients received Neisseria meningitidis meningococcal vaccination | ||
| − | ===Variant # | + | '''26-week course''' |
| + | |||
| + | ===Variant #3, 12-month course {{#subobject:2 |Variant=1}}=== | ||
{| class="wikitable" style="width: 100%; text-align:center;" | {| class="wikitable" style="width: 100%; text-align:center;" | ||
!Study | !Study | ||
| Line 64: | Line 71: | ||
|- | |- | ||
|} | |} | ||
| − | + | ====Therapy==== | |
| − | |||
| − | |||
| − | ==== | ||
*[[Eculizumab (Soliris)]] as follows: | *[[Eculizumab (Soliris)]] as follows: | ||
| − | **600 mg IV over 25 to 45 minutes once per week | + | **Weeks 1 to 4: 600 mg IV over 25 to 45 minutes once per week |
| − | **900 mg IV over 25 to 45 minutes once | + | **Weeks 5 to 52: 900 mg IV over 25 to 45 minutes once every 2 weeks |
| − | ''' | + | '''52-week course''' |
| − | + | ===Variant #4, indefinite {{#subobject:3 |Variant=1}}=== | |
| − | |||
| − | |||
| − | |||
| − | |||
| − | ===Variant # | ||
{| class="wikitable" style="width: 100%; text-align:center;" | {| class="wikitable" style="width: 100%; text-align:center;" | ||
!Study | !Study | ||
| Line 88: | Line 87: | ||
|- | |- | ||
|} | |} | ||
| − | |||
====Initial therapy==== | ====Initial therapy==== | ||
*[[Eculizumab (Soliris)]] as follows: | *[[Eculizumab (Soliris)]] as follows: | ||
| − | **600 mg IV over at least 30 minutes once per day on days 1, 8, 15, 22 | + | **Weeks 1 to 4: 600 mg IV over at least 30 minutes once per day on days 1, 8, 15, 22 |
| − | **900 mg IV over at least 30 minutes once | + | **Week 5 onwards: 900 mg IV over at least 30 minutes once every 2 weeks |
| + | ***Patients with return of PNH symptoms, such as red or black urine, abdominal discomfort, or increased LDH before the next dose of eculizumab received higher doses: [[Eculizumab (Soliris)]] 1200 mg IV once every 2 weeks | ||
| − | + | ====Supportive medications==== | |
| + | *Patients received Neisseria meningitidis meningococcal vaccination | ||
| + | *[[Penicillin V]] 500 mg PO BID as prophylaxis | ||
| + | **Patients with penicillin allergies instead received: [[Erythromycin]] 500 mg PO BID | ||
| − | |||
| − | |||
| − | |||
| − | |||
| − | |||
'''Given indefinitely''' | '''Given indefinitely''' | ||
| − | |||
| − | |||
| − | |||
| − | |||
| − | |||
===References=== | ===References=== | ||
| − | # Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, Cullen MJ, Richards SJ, Rollins SA, Mojcik CF, Rother RP. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004 Feb 5;350(6):552-9. [http://www.nejm.org/doi/full/10.1056/NEJMoa031688 link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/14762182 PubMed] | + | # Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, Cullen MJ, Richards SJ, Rollins SA, Mojcik CF, Rother RP. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004 Feb 5;350(6):552-9. [http://www.nejm.org/doi/full/10.1056/NEJMoa031688 link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/14762182 PubMed] |
## '''Update:''' Hill A, Hillmen P, Richards SJ, Elebute D, Marsh JC, Chan J, Mojcik CF, Rother RP. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood. 2005 Oct 1;106(7):2559-65. Epub 2005 Jun 28. [http://bloodjournal.hematologylibrary.org/content/106/7/2559.long link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/15985537 PubMed] | ## '''Update:''' Hill A, Hillmen P, Richards SJ, Elebute D, Marsh JC, Chan J, Mojcik CF, Rother RP. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood. 2005 Oct 1;106(7):2559-65. Epub 2005 Jun 28. [http://bloodjournal.hematologylibrary.org/content/106/7/2559.long link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/15985537 PubMed] | ||
| − | # Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, Röth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. [http://www.nejm.org/doi/full/10.1056/NEJMoa061648 link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/16990386 PubMed] | + | # '''TRIUMPH:''' Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, Röth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. [http://www.nejm.org/doi/full/10.1056/NEJMoa061648 link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/16990386 PubMed] |
## '''Update:''' Hillmen P, Muus P, Dührsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socié G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. Epub 2007 Aug 16. [http://bloodjournal.hematologylibrary.org/content/110/12/4123.long link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/17702897 PubMed] | ## '''Update:''' Hillmen P, Muus P, Dührsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socié G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. Epub 2007 Aug 16. [http://bloodjournal.hematologylibrary.org/content/110/12/4123.long link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/17702897 PubMed] | ||
| − | # Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, Gaya A, Coyle L, de Castro C, Fu CL, Maciejewski JP, Bessler M, Kroon HA, Rother RP, Hillmen P. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008 Feb 15;111(4):1840-7. Epub 2007 Nov 30. [http://bloodjournal.hematologylibrary.org/content/111/4/1840.long link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/18055865 PubMed] | + | # '''SHEPHERD:''' Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, Gaya A, Coyle L, de Castro C, Fu CL, Maciejewski JP, Bessler M, Kroon HA, Rother RP, Hillmen P. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008 Feb 15;111(4):1840-7. Epub 2007 Nov 30. [http://bloodjournal.hematologylibrary.org/content/111/4/1840.long link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/18055865 PubMed] |
## '''Update:''' Hillmen P, Muus P, Röth A, Elebute MO, Risitano AM, Schrezenmeier H, Szer J, Browne P, Maciejewski JP, Schubert J, Urbano-Ispizua A, de Castro C, Socié G, Brodsky RA. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013 Jul;162(1):62-73. Epub 2013 Apr 25. [https://onlinelibrary.wiley.com/doi/10.1111/bjh.12347/full link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3744747/ link to PMC article] [https://www.ncbi.nlm.nih.gov/pubmed/23617322 PubMed] | ## '''Update:''' Hillmen P, Muus P, Röth A, Elebute MO, Risitano AM, Schrezenmeier H, Szer J, Browne P, Maciejewski JP, Schubert J, Urbano-Ispizua A, de Castro C, Socié G, Brodsky RA. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013 Jul;162(1):62-73. Epub 2013 Apr 25. [https://onlinelibrary.wiley.com/doi/10.1111/bjh.12347/full link to original article] [https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3744747/ link to PMC article] [https://www.ncbi.nlm.nih.gov/pubmed/23617322 PubMed] | ||
# Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, Mitchell LD, Cohen DR, Gregory WM, Hillmen P. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011 Jun 23;117(25):6786-92. Epub 2011 Apr 1. [http://bloodjournal.hematologylibrary.org/content/117/25/6786.long link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/21460245 PubMed] content property of [http://hemonc.org HemOnc.org] | # Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, Mitchell LD, Cohen DR, Gregory WM, Hillmen P. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011 Jun 23;117(25):6786-92. Epub 2011 Apr 1. [http://bloodjournal.hematologylibrary.org/content/117/25/6786.long link to original article] '''contains verified protocol''' [https://www.ncbi.nlm.nih.gov/pubmed/21460245 PubMed] content property of [http://hemonc.org HemOnc.org] | ||
## '''Update: Abstract:''' Anita Hill; Richard J Kelly; Austin G Kulasekararaj; Shreyans A Gandhi; Lindsay D Mitchell; Modupe Elebute; Stephen John Richards; Matthew Cullen; Louise M Arnold; Joanna Large; Alexandra Wood; Gemma L Brooksbank; Tracy Downing; Claire McKinley; Dena Cohen; Walter M Gregory; Judith C. W. Marsh; Ghulam J. Mufti; Peter Hillmen. Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK. 2012 ASH Annual Meeting abstract 3472. [http://abstracts.hematologylibrary.org/cgi/content/abstract/120/21/3472?maxtoshow=&hits=10&RESULTFORMAT=&fulltext=3472&searchid=1&FIRSTINDEX=0&volume=120&issue=21&resourcetype=HWCIT link to abstract] | ## '''Update: Abstract:''' Anita Hill; Richard J Kelly; Austin G Kulasekararaj; Shreyans A Gandhi; Lindsay D Mitchell; Modupe Elebute; Stephen John Richards; Matthew Cullen; Louise M Arnold; Joanna Large; Alexandra Wood; Gemma L Brooksbank; Tracy Downing; Claire McKinley; Dena Cohen; Walter M Gregory; Judith C. W. Marsh; Ghulam J. Mufti; Peter Hillmen. Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK. 2012 ASH Annual Meeting abstract 3472. [http://abstracts.hematologylibrary.org/cgi/content/abstract/120/21/3472?maxtoshow=&hits=10&RESULTFORMAT=&fulltext=3472&searchid=1&FIRSTINDEX=0&volume=120&issue=21&resourcetype=HWCIT link to abstract] | ||
| − | # Kelly RJ, Höchsmann B, Szer J, Kulasekararaj A, de Guibert S, Röth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in | + | # '''Retrospective:''' Kelly RJ, Höchsmann B, Szer J, Kulasekararaj A, de Guibert S, Röth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-1039. [http://www.nejm.org/doi/full/10.1056/NEJMoa1502950 link to original article] [https://www.ncbi.nlm.nih.gov/pubmed/26352814 PubMed] |
==Placebo== | ==Placebo== | ||
Revision as of 02:01, 18 June 2018
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.
| Section editors | |||
|---|---|---|---|
 |
Shruti Chaturvedi, MBBS, MSCI Baltimore, MD |
 |
Benjamin Tillman, MD Nashville, TN |
3 regimens on this page
6 variants on this page
|
All lines of therapy
Eculizumab monotherapy
| back to top |
Variant #1, 12-week course
| Study | Evidence |
|---|---|
| Hillmen et al. 2004 | Pilot, <20 pts |
Therapy
- Eculizumab (Soliris) as follows:
- Weeks 1 to 4: 600 mg IV over 25 to 45 minutes once per week
- Weeks 5 to 12: 900 mg IV over 25 to 45 minutes once every 2 weeks
12-week course
Variant #2, 6-month course
| Study | Evidence | Comparator | Efficacy |
|---|---|---|---|
| Hillmen et al. 2006 (TRIUMPH) | Phase III (E) | Placebo | Superior stabilization of Hgb levels and QoL |
Therapy
- Eculizumab (Soliris) as follows:
- Weeks 1 to 4: 600 mg IV over 25 to 45 minutes once per week
- Weeks 5 to 26: 900 mg IV over 25 to 45 minutes once every 2 weeks
Supportive medications
- Patients received Neisseria meningitidis meningococcal vaccination
26-week course
Variant #3, 12-month course
| Study | Evidence |
|---|---|
| Brodsky et al. 2007 (SHEPHERD) | Phase II |
Therapy
- Eculizumab (Soliris) as follows:
- Weeks 1 to 4: 600 mg IV over 25 to 45 minutes once per week
- Weeks 5 to 52: 900 mg IV over 25 to 45 minutes once every 2 weeks
52-week course
Variant #4, indefinite
| Study | Evidence |
|---|---|
| Kelly et al. 2011 | Non-randomized |
Initial therapy
- Eculizumab (Soliris) as follows:
- Weeks 1 to 4: 600 mg IV over at least 30 minutes once per day on days 1, 8, 15, 22
- Week 5 onwards: 900 mg IV over at least 30 minutes once every 2 weeks
- Patients with return of PNH symptoms, such as red or black urine, abdominal discomfort, or increased LDH before the next dose of eculizumab received higher doses: Eculizumab (Soliris) 1200 mg IV once every 2 weeks
Supportive medications
- Patients received Neisseria meningitidis meningococcal vaccination
- Penicillin V 500 mg PO BID as prophylaxis
- Patients with penicillin allergies instead received: Erythromycin 500 mg PO BID
Given indefinitely
References
- Hillmen P, Hall C, Marsh JC, Elebute M, Bombara MP, Petro BE, Cullen MJ, Richards SJ, Rollins SA, Mojcik CF, Rother RP. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004 Feb 5;350(6):552-9. link to original article contains verified protocol PubMed
- Update: Hill A, Hillmen P, Richards SJ, Elebute D, Marsh JC, Chan J, Mojcik CF, Rother RP. Sustained response and long-term safety of eculizumab in paroxysmal nocturnal hemoglobinuria. Blood. 2005 Oct 1;106(7):2559-65. Epub 2005 Jun 28. link to original article PubMed
- TRIUMPH: Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, Röth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. link to original article contains verified protocol PubMed
- Update: Hillmen P, Muus P, Dührsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socié G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. Epub 2007 Aug 16. link to original article PubMed
- SHEPHERD: Brodsky RA, Young NS, Antonioli E, Risitano AM, Schrezenmeier H, Schubert J, Gaya A, Coyle L, de Castro C, Fu CL, Maciejewski JP, Bessler M, Kroon HA, Rother RP, Hillmen P. Multicenter phase 3 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008 Feb 15;111(4):1840-7. Epub 2007 Nov 30. link to original article contains verified protocol PubMed
- Update: Hillmen P, Muus P, Röth A, Elebute MO, Risitano AM, Schrezenmeier H, Szer J, Browne P, Maciejewski JP, Schubert J, Urbano-Ispizua A, de Castro C, Socié G, Brodsky RA. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013 Jul;162(1):62-73. Epub 2013 Apr 25. link to original article link to PMC article PubMed
- Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, Mitchell LD, Cohen DR, Gregory WM, Hillmen P. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011 Jun 23;117(25):6786-92. Epub 2011 Apr 1. link to original article contains verified protocol PubMed content property of HemOnc.org
- Update: Abstract: Anita Hill; Richard J Kelly; Austin G Kulasekararaj; Shreyans A Gandhi; Lindsay D Mitchell; Modupe Elebute; Stephen John Richards; Matthew Cullen; Louise M Arnold; Joanna Large; Alexandra Wood; Gemma L Brooksbank; Tracy Downing; Claire McKinley; Dena Cohen; Walter M Gregory; Judith C. W. Marsh; Ghulam J. Mufti; Peter Hillmen. Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK. 2012 ASH Annual Meeting abstract 3472. link to abstract
- Retrospective: Kelly RJ, Höchsmann B, Szer J, Kulasekararaj A, de Guibert S, Röth A, Weitz IC, Armstrong E, Risitano AM, Patriquin CJ, Terriou L, Muus P, Hill A, Turner MP, Schrezenmeier H, Peffault de Latour R. Eculizumab in pregnant patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2015 Sep 10;373(11):1032-1039. link to original article PubMed
Placebo
| back to top |
Regimen
| Study | Evidence | Comparator | Efficacy |
|---|---|---|---|
| Hillmen et al. 2006 (TRIUMPH) | Phase III | Eculizumab | Inferior stabilization of Hgb levels and QoL |
No active treatment. Used as a comparator arm in one or more trials; for reference purposes, only.
References
- Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, Röth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006 Sep 21;355(12):1233-43. link to original article contains verified protocol PubMed
- Update: Hillmen P, Muus P, Dührsen U, Risitano AM, Schubert J, Luzzatto L, Schrezenmeier H, Szer J, Brodsky RA, Hill A, Socié G, Bessler M, Rollins SA, Bell L, Rother RP, Young NS. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007 Dec 1;110(12):4123-8. Epub 2007 Aug 16. link to original article PubMed