Diagnosis guide for clinicians
Paroxysmal nocturnal hemoglobinuria (PNH): when to suspect it and what to test
Paroxysmal nocturnal hemoglobinuria (PNH) is a nonmalignant clonal expansion of hematopoietic stem cells with an acquired PIGA mutation, whose progeny lack GPI-anchored proteins such as the complement regulators CD55 and CD59.International PNH Interest Group
The International PNH Interest Group names hemolytic anemia, marrow failure and thrombophilia as the primary clinical manifestations, and notes that PNH frequently arises with aplastic anemia.International PNH Interest Group
A Canadian PNH Network consensus says the variety of ways patients present can lead to significant delays in recognition.Canadian PNH Network consensus
Red flags for Paroxysmal nocturnal hemoglobinuria (PNH)
What the cited sources describe, grouped by life stage and organ. Each line links to its source.
Hemolysis and anemia
- Coombs-negative hemolytic anemia is the clinical hallmark of PNH, according to the International PNH Interest Group.International PNH Interest Group
- Classic PNH shows intravascular hemolysis: reticulocytosis, high LDH and indirect bilirubin, and low haptoglobin.International PNH Interest Group
- The Interest Group screening list includes Coombs-negative intravascular hemolysis, based on a high LDH, especially with concurrent iron deficiency; urinary iron loss is high in PNH and iron deficiency is common.International PNH Interest Group
- Hemoglobinuria, with urine ranging from dark tea to black to cherry red.Nature Reviews Disease Primers
- Disabling fatigue that can be disproportionate to the degree of anemia, often most intense during a hemolytic attack.Nature Reviews Disease Primers
- Jaundice and hemoglobinuria are often exacerbated by infections, surgery, exercise, pregnancy or excessive alcohol intake.Nature Reviews Disease Primers
Thrombosis at unusual sites
- The Interest Group lists venous thrombosis in unusual sites as a screening indication: Budd-Chiari syndrome, mesenteric or portal veins, cerebral veins and dermal veins.International PNH Interest Group
- Hill and colleagues call hepatic vein thrombosis the most common; portal, mesenteric, splenic and cerebral sinus veins are other frequent sites.Nature Reviews Disease Primers
- The Interest Group says patients with thrombosis at unusual sites should be screened, especially with coexisting cytopenias, intravascular hemolysis or both.International PNH Interest Group
- Deep vein thrombosis, pulmonary emboli and dermal thrombosis are also relatively common; venous thrombosis tends to be more common than arterial.Nature Reviews Disease Primers
- In a retrospective review of pregnancy in women with PNH, cited by the Interest Group, PNH was first diagnosed during pregnancy in 25% of cases.International PNH Interest Group
Swallowing, abdominal pain and erectile dysfunction
- Episodic dysphagia or abdominal pain with evidence of intravascular hemolysis is on the Interest Group screening list.International PNH Interest Group
- Dysphagia and odynophagia, which appear to come from esophageal spasm, especially during hemolytic exacerbations.International PNH Interest Group
- Gastrointestinal complaints may be the first manifestation in about 10% of patients.International PNH Interest Group
- Recurrent colicky abdominal pain; mesenteric vein thrombosis can cause intestinal infarction.International PNH Interest Group
- Erectile dysfunction, reported by 38% of men among 856 patients in International PNH Registry baseline data.Nature Reviews Disease Primers
Cytopenias and marrow failure
- Aplastic anemia: the Interest Group recommends screening at diagnosis and at least yearly, even without evidence of hemolysis.International PNH Interest Group
- Refractory anemia-MDS is also on the Interest Group screening list, even without clinical evidence of hemolysis.International PNH Interest Group
- Hill and colleagues say flow cytometry should be requested for aplastic anemia or unexplained pancytopenia.Nature Reviews Disease Primers
- A 2023 review says PNH should be considered with any otherwise unexplained cytopenia, aplastic anemia or other marrow failure, atypical thrombosis, Coombs-negative hemolysis or hemoglobinuria.Journal of Blood Medicine review
Kidney and lungs
- Long-term intravascular hemolysis raises the risk of chronic kidney disease, with tubular damage from microvascular thrombosis, iron deposits or both.Nature Reviews Disease Primers
- Acute renal failure can occur; Hill and colleagues recommend Doppler ultrasound to rule out renal vein thrombosis.Nature Reviews Disease Primers
- Mild to moderate pulmonary hypertension has been reported in PNH.Nature Reviews Disease Primers
Often mistaken for
Diagnoses patients commonly carry first, and why the sources say they are confused.
| First diagnosis | Why it is confused |
|---|---|
| Aplastic anemia | PNH frequently arises with aplastic anemia, and 10 of 80 patients in a series cited by the Interest Group presented with it; the two can coexist.International PNH Interest Group |
| Myelodysplastic syndrome | Erythroid dysplasia from rapid red cell turnover can make the marrow resemble MDS, and karyotypic abnormalities might be present in up to 25% of patients.Nature Reviews Disease Primers |
| Iron deficiency anemia | Chronic hemoglobinuria and hemosiderinuria drain iron, and 5 of 80 patients in the cited series presented with iron deficiency anemia.International PNH Interest Group |
| Unexplained venous thrombosis or Budd-Chiari syndrome | Thrombosis or embolism was the presenting feature in 5 of 80 patients, and hepatic, mesenteric, portal, cerebral and dermal veins are disproportionately represented among PNH clots.International PNH Interest Group |
| Esophageal spasm or a swallowing disorder | Dysphagia and odynophagia in PNH appear to come from esophageal spasm, especially during hemolytic exacerbations.International PNH Interest Group |
| Recurrent abdominal pain of unclear cause | Some patients have recurrent colicky abdominal pain, and thrombosis of mesenteric vessels appears to play a role in some cases.International PNH Interest Group |
| Erectile dysfunction | Males with PNH may have episodes of impotence, particularly during hemolytic exacerbations.International PNH Interest Group |
Codes patients often carry before this diagnosis
For spotting the pattern in a problem list or registry. Descriptions are checked daily against NLM's ICD-10-CM service; this is not coding advice.
I82.0Hepatic vein thrombosis is the most common thrombosis site in PNH, per Hill and colleagues.Nature Reviews Disease Primers
I81Portal vein thrombosis is among the intra-abdominal sites on the Interest Group screening list.International PNH Interest Group
I67.6, G08Cerebral vein thrombosis is among the unusual-site thromboses on the Interest Group screening list.International PNH Interest Group
I82.3Hill and colleagues recommend ruling out renal vein thrombosis in acute renal failure in PNH.Nature Reviews Disease Primers
D59.9Coombs-negative hemolytic anemia is the clinical hallmark of PNH.International PNH Interest Group
R82.3Hemoglobinuria is on the Interest Group screening list.International PNH Interest Group
D50.9Urinary iron loss makes iron deficiency common, and some patients present with it.International PNH Interest Group
D61.3, D61.9The Interest Group recommends PNH screening in aplastic anemia at diagnosis and at least yearly.International PNH Interest Group
D46.0, D46.4, D46.9Refractory anemia-MDS is on the Interest Group screening list.International PNH Interest Group
D61.818Hill and colleagues say flow cytometry should be requested for unexplained pancytopenia.Nature Reviews Disease Primers
R13.10, R13.19Episodic dysphagia with intravascular hemolysis is on the Interest Group screening list.International PNH Interest Group
What to test, step by step
The testing order the cited guidance describes, by who it applies to. It summarizes published guidance and is not a plan for any one patient.
Suspected PNH
- Blood count and hemolysis markers
The International PNH Interest Group lists a complete blood count, reticulocyte count, LDH, fractionated bilirubin and haptoglobin among its minimal criteria for diagnosis and categorization.International PNH Interest Group
- Peripheral blood flow cytometry for GPI-anchored proteins
The Interest Group calls flow cytometry the most sensitive and informative diagnostic assay. It recommends measuring at least 2 GPI-anchored proteins at first testing, to exclude an inherited deficiency of a single protein.International PNH Interest Group
- At least two cell lineages
Hill and colleagues say the diagnosis should be confirmed by absent or severely deficient GPI-anchored proteins on at least two blood cell lineages.Nature Reviews Disease Primers
- High-sensitivity analysis
The 2010 ICCS guidelines separate routine analysis, which identifies abnormal populations of 1% or more, from high-sensitivity analysis, which detects as few as 0.01% PNH cells.ICCS 2010 guidelines
Patients with hemolysis
- Direct antiglobulin (Coombs) test
Hill and colleagues list Coombs-negative hemolytic anemia among the reasons to request PNH flow cytometry.Nature Reviews Disease Primers
Granulocytes and monocytes
- FLAER-based white cell assay
A 2014 Cytometry B study notes that flow guidelines recommend FLAER-based assays for granulocytes and monocytes. It tested CD157 as a replacement for CD24 on granulocytes and CD14 on monocytes.CD157 FLAER assay study
Red cells
- CD235a and CD59 red cell assay
ICCS-based practical guidelines describe a CD235a/CD59 assay that detected as few as 20 type III PNH red cells per million. In 10 normal samples, type III cells ranged from 0 to 6 per million.ICCS-based practical guidelines
Anyone with a PNH clone
- Interpreting a positive result
The 2018 ICCS/ESCCA guidelines note that an abnormal flow result is not diagnostic of PNH by itself. Laboratory and clinical features together establish the diagnosis, and clone size and cell types help classify it.ICCS/ESCCA 2018, Part 1
Confirmed PNH clone
- Classification
The Interest Group classifies disease as classic PNH, PNH with another marrow disorder such as aplastic anemia or refractory anemia-MDS, or subclinical PNH, with small clones and no clinical or laboratory evidence of hemolysis, alongside another marrow disorder.International PNH Interest Group
- Bone marrow aspirate, biopsy and cytogenetics
The Interest Group lists marrow aspirate, biopsy and cytogenetics among its minimal criteria, because marrow analysis is required to assign the subcategory.International PNH Interest Group
- Bone marrow: a differing view
Hill and colleagues write that marrow biopsy is not required for the diagnosis of PNH but should be performed in patients with severe pancytopenia.Nature Reviews Disease Primers
Confirmed PNH
- Supporting studies
The Interest Group recommends iron studies, creatinine and BUN, serum erythropoietin and urine hemosiderin. It pairs them with a history focused on hemoglobinuria, thrombosis, dysphagia, abdominal pain and impotence.International PNH Interest Group
Patients with confirmed PNH
- Label scope: eculizumab (Soliris)
The Soliris label indicates it for patients with PNH to reduce hemolysis. Because of the risk of serious meningococcal infections, it is available only through a restricted program, the ULTOMIRIS and SOLIRIS REMS.Soliris label
- Label scope: eculizumab-aeeb (Bkemv)
The Bkemv label, for a biosimilar to Soliris, carries the same PNH indication, to reduce hemolysis. Because of the risk of serious meningococcal infections, it is available only through the BKEMV REMS.Bkemv label
- Label scope: eculizumab-aagh (Epysqli)
The Epysqli label, for another biosimilar to Soliris, also covers PNH to reduce hemolysis. Because of the risk of serious meningococcal infections, it is available only through the EPYSQLI REMS.Epysqli label
Adults and children 1 month and older with PNH
- Label scope: ravulizumab (Ultomiris)
The Ultomiris label covers adult and pediatric patients one month of age and older with PNH. Because of the risk of serious meningococcal infections, it is available only through the ULTOMIRIS and SOLIRIS REMS.Ultomiris label
Patients 13 and older weighing at least 40kg
- Label scope: crovalimab (Piasky)
The Piasky label covers adult and pediatric patients 13 years and older with PNH and a body weight of at least 40kg. Because of the risk of serious meningococcal infections, it is available only through the PIASKY REMS.Piasky label
Adults with PNH
- Label scope: pegcetacoplan (Empaveli)
The Empaveli label covers adult patients with PNH. Because of the risk of serious infections caused by encapsulated bacteria, it is available only through the EMPAVELI REMS.Empaveli label
- Label scope: iptacopan (Fabhalta)
The Fabhalta label covers adults with PNH. Because of the risk of serious infections caused by encapsulated bacteria, it is available only through the FABHALTA REMS.Fabhalta label
Adults with PNH and extravascular hemolysis
- Label scope: danicopan (Voydeya)
The Voydeya label covers add-on therapy to ravulizumab or eculizumab for extravascular hemolysis in adults with PNH, and says it has not been shown to be effective as monotherapy. Because of the risk of serious infections caused by encapsulated bacteria, it is available only through the VOYDEYA REMS.Voydeya label
Where testing goes wrong
- Only 26% of 80 patients in a cited series reported hemoglobinuria at presentation; it may be absent with PNH and aplastic anemia or refractory anemia-MDS, where clones are often small.International PNH Interest Group
- GARD notes that hemoglobinuria is most noticeable early in the morning in many, but not all, cases.GARD
- Red cell transfusion raises the share of normal red cells. For an accurate red cell percentage, the Interest Group advises analysis before transfusion or after at least 1 month without one, longer if clinically safe.International PNH Interest Group
- Granulocytes have a normal life span, so their GPI-deficient share more accurately reflects clone size and is unaffected by red cell transfusion.International PNH Interest Group
- Hill and colleagues write that FLAER does not stain red cells, because red cells express high levels of glycophorin, which binds aerolysin and interferes with the assay.Nature Reviews Disease Primers
- The Interest Group wrote in 2005 that standard single-color flow cytometry is sensitive enough to detect 3% GPI-deficient cells accurately, and that not all laboratories can perform high-sensitivity analysis.International PNH Interest Group
- The Ham test and sucrose lysis test have largely been abandoned, because both are less sensitive and less quantitative than flow cytometry.International PNH Interest Group
- Tiny PNH populations under 0.01% occur in some healthy people and in MDS; Hill and colleagues call them not clinically or diagnostically significant.Nature Reviews Disease Primers
- Patients with mostly type II PNH red cells could have a large clone with only subtle clinical evidence of hemolysis.International PNH Interest Group
- A thromboembolic event is the presenting feature in about 5% of patients, and the Interest Group does not recommend routine PNH screening of all patients with thrombosis.International PNH Interest Group
- The Interest Group calls screening for clinical forms of PNH unwarranted without a raised LDH, but says this criterion does not apply to subclinical PNH, which by definition lacks clinical evidence of hemolysis.International PNH Interest Group
- In an Italian study of 583 clones, 13 involved only white cells and 3 involved only red cells.Italian multicenter clone study
Where to test
- Flow cytometry laboratory with high-sensitivity PNH testing: To diagnose and follow subclinical PNH, the International PNH Interest Group encourages physicians to identify laboratories that can consistently and reproducibly detect less than 1.0% GPI-deficient red cells and granulocytes.International PNH Interest Group
- ICCS/ESCCA 2018 guidelines, Part 2: Covers instrument setup, antibody conjugate selection and assay design for high-sensitivity PNH testing of red cells, neutrophils and monocytes.ICCS/ESCCA 2018, Part 2
Family screening and referral
Who else in the family
- MedlinePlus Genetics describes PNH as acquired rather than inherited, caused in almost all cases by PIGA variants that arise during life and are present only in certain cells.MedlinePlus Genetics
- The same source says this form generally occurs with no family history and is not passed down to children.MedlinePlus Genetics
- A very rare inflammatory form involves an inherited altered PIGT copy plus a second, acquired change in the other copy; MedlinePlus says the risk follows an autosomal dominant pattern.MedlinePlus Genetics
- For follow-up, the Interest Group recommends high-sensitivity flow cytometry in aplastic anemia and refractory anemia-MDS without clinical evidence of hemolysis, at diagnosis and every year.International PNH Interest Group
- For PNH with clinical evidence of hemolysis, it recommends flow cytometry every 6 months for 2 years, then yearly if stable, and sooner with clinical progression or improvement.International PNH Interest Group
- Clones in aplastic anemia can expand, and patients may later meet criteria for hemolytic PNH; clone expansion may accompany aplastic anemia relapse.Nature Reviews Disease Primers
- Over 1 year of follow-up retesting in 151 Italian cases, clone size rose markedly in 4 and fell in 13.Italian multicenter clone study
- Hill and colleagues list evolution to myelodysplastic syndromes among the clinical manifestations of PNH.Nature Reviews Disease Primers
- GARD notes an increased risk of leukemia, and that signs and symptoms disappear on their own in a small number of patients.GARD
Who to refer to
- GARD lists hematologists among the specialists who may help diagnose and treat PNH, and links to the American Society of Hematology directory.GARD
- GARD names the Aplastic Anemia and MDS International Foundation among patient organizations that may maintain a list of experts.GARD
- The 2018 ICCS/ESCCA guidelines say the ordering clinician needs to understand the test, the disease biology and the patient's clinical picture to use the result.ICCS/ESCCA 2018, Part 1
- A 2021 ASH Education Program review reports that only about one-third of patients with PNH clones have classical PNH; two-thirds of PNH clones occur in patients who present with marrow failure and few, if any, PNH symptoms.ASH Education Program review
- For pregnancy, the Interest Group advises a team with an experienced hematologist and an obstetrician who specializes in high-risk pregnancy.International PNH Interest Group
- A 2023 review calls allogeneic stem cell transplant the only curative therapy and says it is reserved for PNH with severe aplastic anemia or other marrow failure, because transplant risks are significant.Journal of Blood Medicine review
Find a specialist by state
From the CMS NPI Registry, a public directory of US clinicians by registered specialty. A listing is not an endorsement and says nothing about experience with Paroxysmal nocturnal hemoglobinuria (PNH); patient organizations and academic centers can often name experienced clinicians.
ICD-10-CM codes for Paroxysmal nocturnal hemoglobinuria (PNH)
For orders, referrals and prior authorization. Each code is checked daily against NLM's ICD-10-CM service.
Once the diagnosis is confirmed
For the patient: why your doctor ordered this test
Plain language a patient can read or take home.
Your doctor ordered this test to check for a rare blood condition called PNH.
In PNH, some blood cells are missing a protective coating. Without it, the body's own defenses can break open red blood cells.
This can cause tiredness, dark urine, belly pain, trouble swallowing or blood clots.
The test uses a regular blood sample. A lab checks your blood cells for the missing coating with a method called flow cytometry.
A recent blood transfusion can change the red blood cell results. Tell your doctor if you have had one. The lab also checks white blood cells, which a red blood cell transfusion does not change.
PNH is almost always acquired during life, not inherited. The usual form is not passed from parents to children.
If you have aplastic anemia, your doctor may repeat this test about once a year.
Questions clinicians ask about Paroxysmal nocturnal hemoglobinuria (PNH) testing
What test confirms paroxysmal nocturnal hemoglobinuria?
Peripheral blood flow cytometry showing blood cells that lack GPI-anchored proteins is the diagnostic test, per the International PNH Interest Group (Blood, 2005). The group calls it the most sensitive and informative assay and recommends measuring at least 2 GPI-anchored proteins at first testing. Hill and colleagues (2017) describe confirmation on at least two cell lineages, using antibodies such as anti-CD59 or the FLAER reagent. The 2018 ICCS/ESCCA guidelines add that an abnormal result is read together with clinical and laboratory features.
Who should be screened for PNH?
The International PNH Interest Group lists people with hemoglobinuria, Coombs-negative intravascular hemolysis, venous thrombosis at unusual sites, aplastic anemia, refractory anemia-MDS, or episodic dysphagia or abdominal pain with hemolysis. It recommends screening aplastic anemia at diagnosis and at least yearly, even without hemolysis. It does not recommend routine screening of all patients with thrombosis.
Does a patient need dark urine to have PNH?
No; only 26% of 80 patients in a series cited by the International PNH Interest Group reported hemoglobinuria at presentation. The group notes it may be absent when the clone is small, as in PNH with aplastic anemia or refractory anemia-MDS. GARD says hemoglobinuria is most noticeable early in the morning in many, but not all, cases.
Can PNH flow cytometry be done after a blood transfusion?
Yes; the International PNH Interest Group says recent transfusion is unlikely to obscure the diagnosis, though it raises the share of normal red cells. Granulocyte results are unaffected by red cell transfusion and more accurately reflect clone size. For an accurate red cell percentage, the group advises testing before transfusion or after at least 1 month without one, longer if clinically safe.
Is PNH inherited?
Almost never; MedlinePlus Genetics describes PNH as acquired, caused in almost all cases by PIGA variants that arise during life in blood-forming stem cells and are not passed to children. A very rare inflammatory form involves an inherited PIGT variant plus a second, acquired change. The Interest Group separately recommends yearly high-sensitivity flow cytometry for patients with aplastic anemia without clinical evidence of hemolysis.
Which FDA labels cover PNH?
DailyMed and openFDA labels checked November 9, 2026 list Soliris (eculizumab) and its biosimilars Bkemv and Epysqli for PNH to reduce hemolysis. Ultomiris (ravulizumab) covers adults and children 1 month and older, Empaveli (pegcetacoplan) and Fabhalta (iptacopan) cover adults, and Piasky (crovalimab) covers patients 13 and older weighing at least 40kg. Voydeya (danicopan) is labeled only as add-on therapy to ravulizumab or eculizumab for extravascular hemolysis in adults. All eight labels say the drug is available only through a REMS because of the risk of serious infections.
What is the ICD-10 code for PNH?
The ICD-10-CM code is D59.5, paroxysmal nocturnal hemoglobinuria [Marchiafava-Micheli], per the National Library of Medicine's ICD-10-CM lookup checked November 9, 2026. The lookup does not say which code fits a given patient; that is a coding decision.
Sources
- Diagnosis and management of paroxysmal nocturnal hemoglobinuria. Blood 2005;106(12):3699-3709 (Parker C et al for the International PNH Interest Group; supported in part by Japanese government and foundation grants, an NIH grant, the Aplastic Anemia and MDS International Foundation and the HPN France patient association; the Interest Group member list includes six Alexion Pharmaceuticals employees), via PubMed Central, 2005-12-01.
- Paroxysmal nocturnal haemoglobinuria. Nature Reviews Disease Primers 2017;3:17028 (Hill A, DeZern AE, Kinoshita T, Brodsky RA; supported in part by the Aplastic Anemia and MDS International Foundation and NIH. Hill reports honoraria or consulting from Akari, Alexion, Alnylam, Ra Pharmaceuticals and Roche; Kinoshita from Alexion; Brodsky sits on Alexion, Apellis and Achillion advisory boards and receives Alexion grant funding; DeZern reports none), author manuscript via PubMed Central, 2017-05-18.
- The incidence and prevalence of patients with paroxysmal nocturnal haemoglobinuria and aplastic anaemia PNH syndrome: A retrospective analysis of the UK's population-based haematological malignancy research network 2004-2018. European Journal of Haematology 2021;107(2):211-218 (Richards SJ et al; one author lists an Alexion Pharmaceuticals affiliation; the source Orphanet cites for its UK figures), via PubMed abstract, 2021-06-09.
- ICCS/ESCCA consensus guidelines to detect GPI-deficient cells in paroxysmal nocturnal hemoglobinuria (PNH) and related disorders part 1 - clinical utility. Cytometry Part B: Clinical Cytometry 2018;94(1):16-22 (Dezern AE, Borowitz MJ; International Clinical Cytometry Society), via PubMed abstract; funding and disclosures not seen, 2018-01.
- ICCS/ESCCA consensus guidelines to detect GPI-deficient cells in paroxysmal nocturnal hemoglobinuria (PNH) and related disorders part 2 - reagent selection and assay optimization for high-sensitivity testing. Cytometry Part B: Clinical Cytometry 2018;94(1):23-48 (Sutherland DR et al; International Clinical Cytometry Society), via PubMed abstract; funding and disclosures not seen, 2018-01.
- Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometry. Cytometry Part B: Clinical Cytometry 2010;78(4):211-230 (Borowitz MJ et al), via PubMed abstract; funding and disclosures not seen, 2010-07.
- Practical guidelines for the high-sensitivity detection and monitoring of paroxysmal nocturnal hemoglobinuria clones by flow cytometry. Cytometry Part B: Clinical Cytometry 2012;82(4):195-208 (Sutherland DR, Keeney M, Illingworth A), via PubMed abstract; funding and disclosures not seen, 2012-04-25.
- Use of CD157 in FLAER-based assays for high-sensitivity PNH granulocyte and PNH monocyte detection. Cytometry Part B: Clinical Cytometry 2014;86(1):44-55 (Sutherland DR et al), via PubMed abstract; funding and disclosures not seen, 2013-08-06.
- Features, reason for testing, and changes with time of 583 paroxysmal nocturnal hemoglobinuria clones from 529 patients: a multicenter Italian study. Annals of Hematology 2019;98(5):1083-1093 (Cannizzo E et al), via PubMed abstract; funding and disclosures not seen, 2019-03-13.
- How we treat paroxysmal nocturnal hemoglobinuria: A consensus statement of the Canadian PNH Network and review of the national registry. European Journal of Haematology 2019;102(1):36-52 (Patriquin CJ et al), via PubMed abstract; funding and disclosures not seen, 2018-10-25.
- When does a PNH clone have clinical significance?. Hematology: the American Society of Hematology Education Program 2021;2021(1):143-152 (Babushok DV; supported by NHLBI grant K08 HL132101; the author declares no competing financial interests), via PubMed Central, 2021-12-10.
- Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations. Journal of Blood Medicine 2023;14:613-628 (Oliver M, Patriquin CJ; outside the submitted work, Oliver reports personal fees from Alexion, Sobi, Novartis, Takeda and Sanofi, and Patriquin from Alexion, BioCryst, Amgen, Roche, Novartis, Sobi, Sanofi and Takeda and was global PI of a Regeneron PNH trial), via PubMed Central, 2023-12-06.
- Paroxysmal nocturnal hemoglobinuria | About the Disease | GARD. Genetic and Rare Diseases Information Center, NIH, 2026-09.
- Paroxysmal nocturnal hemoglobinuria: MedlinePlus Genetics. National Library of Medicine, 2022-02-24.
- SOLIRIS- eculizumab injection, solution, concentrate. Alexion Pharmaceuticals, via DailyMed, 2026-07-08.
- BKEMV- eculizumab-aeeb injection, solution, concentrate. Amgen, via DailyMed, 2026-07-02.
- EPYSQLI- eculizumab-aagh injection, solution. Teva Pharmaceuticals USA, via DailyMed, 2026-07-06.
- ULTOMIRIS- ravulizumab solution, concentrate. Alexion Pharmaceuticals, via DailyMed, 2026-10-29.
- EMPAVELI- pegcetacoplan injection, solution. Apellis Pharmaceuticals, via DailyMed, 2026-08-21.
- FABHALTA- iptacopan capsule. Novartis Pharmaceuticals, via DailyMed, 2026-07-16.
- PIASKY- crovalimab injection, solution. Genentech, via DailyMed, 2025-11-11.
- VOYDEYA- danicopan tablet, film coated. Alexion Pharmaceuticals, via DailyMed, 2026-05-12.
- ICD-10-CM search results for "paroxysmal nocturnal" (Clinical Table Search Service). National Library of Medicine, 2026-11-09.
A reference for licensed professionals, checked against its sources on November 9, 2026. It summarizes what the cited guidance says and does not tell anyone how to diagnose or treat a specific patient. Guidelines change; the current guideline and the treating clinician's judgment govern. Trial Friend is an independent information service, not a medical provider.