The first European study into the burden of non-tuberculosis mycobacterium pulmonary disease (NTM-PD), published today in the European Respiratory Journal, has revealed that both the 3-year all-cause mortality rate and financial burden for patients with the disease is almost four times higher than a matched control group.1
The study compared 125 newly diagnosed NTM-PD patients with 1250 control patients matched by age, gender and Charlson Comorbidity Index*over a period of 39 months. During this time period the all-cause mortality rate for those diagnosed with NTM-PD was 22.4%, almost four times higher than the matched control group (6%) (P<0.001).1 In the same observation period, the all-cause mortality rate in COPD patients who had NTM-PD was 41.5% (27/65 patients) but was only 15.9% (62/390) in COPD patients without NTM-PD (P<0.001).1
The mean direct expenditure for an NTM-PD patient was €39,559.60 over 39 months, also almost four times higher than the matched control group (€10,006.71).1 Hospitalisations were three times higher for NTM-PD patients and accounted for the majority of expenditure (63%), with drug costs being the second biggest costs.1 Also notable was the discrepancy across individual treatments—only 54% of patients received antibiotics on diagnosis, 26% never received antibiotics at all, and 29 different drug combinations were observed across all treatments.1 Clarithromycin combined with ethambutol was the most frequently prescribed treatment option in 24/93, or 25.8%, of the treated patients, followed by standard treatment clarithromycin plus rifampicin plus ethambutol or rifabutin plus ethambutol in 18/93 patients (19.4%) and 14/93 patients (15.1%), respectively.1
“Although NTM-PD is considered rare, these findings highlight that the burden of the disease is high,” commented Dr Michael Loebinger of the Royal Brompton Hospital, London and one of the study’s authors. “Treatment can be hard to tolerate and may be unsuccessful, making treatment decisions complex. To help with this challenge, the British Thoracic Society will be releasing new guidelines this year.”
NTM-PD is a rare but emerging global health concern, with important public health implications.2,3 The infection is caused by ubiquitous mycobacteria found in the soil and water.3 Over 150 species of NTM have been identified and mycobacterium avium complex (MAC) has been reported to be the most common causative agent in NTM-PD worldwide.3 Diseases caused by NTMs represent a growing diagnostic and clinical challenge.4 In recent decades, the incidence, prevalence, hospitalisation rate, and mortality rate of NTM-PD have increased worldwide.4
NTMs represent a risk of infection for susceptible individuals – these are often patients with a predisposing chronic pulmonary disease, such as COPD or bronchiectasis.4 For these patients, the symptoms of the underlying disease overlap with the non-specific symptoms of NTM-PD, often leading to correct diagnosis often only being made at a late stage.4 From a therapeutic perspective, NTM-PD is more challenging than uncomplicated pulmonary tuberculosis: treatment is lengthy, toxic and often fails.4 Initial treatment has the highest chance of success.4
Insmed Incorporated is a global biopharmaceutical company focused on the unmet needs of patients with rare diseases. The company is advancing a global phase 3 clinical study of ARIKAYCE (liposomal amikacin for inhalation) for adult patients with treatment refractory nontuberculous mycobacteria (NTM) lung disease caused by Mycobacterium avium complex (MAC), a rare and often chronic infection that is capable of causing irreversible lung damage and can be fatal. There are currently no approved inhaled products specifically indicated for the treatment of refractory NTM lung infections caused by MAC in the United States or European Union (EU). Insmed's earlier-stage clinical pipeline includes INS1007, a novel oral reversible inhibitor of DPP1 with therapeutic potential in non-CF bronchiectasis, and INS1009, an inhaled nanoparticle formulation of a treprostinil prodrug that may offer a differentiated product profile for rare disorders, including pulmonary arterial hypertension (PAH). For more information, visit www.insmed.com.
"Insmed" and "ARIKAYCE" are the company's trademarks. All other trademarks, trade names or service marks appearing in this press release are the property of their respective owners.
1 Diel R, Jacob J, Lampenius N et al. Burden of non-tuberculous mycobacterial pulmonary disease in Germany. European Respiratory Journal 2017; 49: 1602109
2 Adjemian J, Olivier K, Seitz A et al. Spatial Clusters of Nontuberculous Mycobacterial Lung Disease in the United States. American Journal of Respiratory and Critical Care Medicine 2012; 185(8): 881-886
3 Johnson MM, Odell JA. Nontuberculous mycobacterial pulmonary infections. Journal of Thoracic Disease 2014; 6(3): 210-220
4 Ringshausen FC, Rademacher J. Lungenerkrankungen durch nichttuberkulöse Mykobakterien. [Pulmonary disease caused by nontuberculous mycobacteria]. Internist 2016; 57: 142–152
* Charlson Comorbidity Index is a measure of 1-year mortality risk and burden of disease, used to assist health professionals to stratify patients into subgroups based on disease severity.